No matter what type of health insurance your patient has, they may have options to help them afford their medicine. Options may be available to your patient even if they have no insurance at all.
Use our financial assistance tool to see which programs may be right for your patient.
If you would rather talk through some potential options, call us at 866-4ACCESS (866-422-2377) (9AM-8PM ET, Monday through Friday).
Quick Links
Help With Co-pay Costs
These programs help your patient pay for Evrysdi if they have insurance but still need help with costs:
Help With Costs for Evrysdi
With the Evrysdi Co-pay Program, eligible patients with commercial insurance could pay as little as $0 per 30-day supply of Evrysdi. The rest of your co-pay or co-insurance is covered, up to $25,000 in assistance per calendar year.
Your patient may be eligible if they:
- Are taking Evrysdi for an FDA-approved use
- Are 18 years of age or older or have a Legally Authorized Person over the age of 18 to manage the program
- Have commercial (private or non-governmental) insurance. This includes plans available through state and federal health insurance exchanges
- Live and receive treatment in the United States or U.S. Territories
- Are not receiving assistance through the Genentech Patient Foundation or any other charitable organization for the same expenses covered by the program
- Do not use a state or federal healthcare plan to pay for your medication. This includes, but is not limited to, Medicare, Medicaid and TRICARE
The Co-pay Program (“Program”) is valid ONLY for patients with commercial (private or non-governmental) insurance who have a valid prescription for a Food and Drug Administration (FDA)-approved indication of a Genentech medicine. The Program is not available to patients whose prescriptions are reimbursed under any federal, state, or government-funded insurance programs (included but not limited to Medicare, Medicare Advantage, Medigap, Medicaid, TRICARE, Department of Defense, or Veterans Affairs Programs) or where prohibited by law or by the patient's health insurance provider. If at any time a patient begins receiving prescription drug coverage under any such federal, state or government-funded healthcare programs, the patient will no longer be eligible for the Program.
Under the Program, the patient may be required to pay a co-pay. The final amount owed by a patient may be as little as $0 for the Genentech medicine (see Program specific details available at the Program website). The total patient out-of-pocket cost is dependent on the patient’s health insurance plan. The Program assists with the cost of the Genentech medicine only. It does not assist with the cost of other medicines, procedures or office visit fees. After reaching the maximum annual Program benefit amount, the patient will be responsible for all remaining out-of-pocket expenses. The Program benefit amount cannot exceed the patient’s out-of-pocket expenses for the Genentech medicine.
All participants are responsible for reporting the receipt of all Program benefits as required by any insurer or by law. The Program is only valid in the United States and U.S. Territories, is void where prohibited by law and shall follow state restrictions in relation to AB-rated generic equivalents (e.g., MA, CA) where applicable. No party may seek reimbursement for all or any part of the benefit received through the Program. The value of the Program is intended exclusively for the benefit of the patient. The funds made available through the Program may only be used to reduce the out-of-pocket costs for the patient enrolled in the Program. The Program is not intended for the benefit of third parties, including without limitation third party payers, pharmacy benefit managers, or their agents. If Genentech determines that a third party has implemented a program that adjusts patient cost-sharing obligations based on the availability of support under the Program and/or excludes the assistance provided under the Program from counting towards the patient’s deductible or out-of-pocket cost limitations, Genentech may impose a per fill cap on the cost-sharing assistance available under the Program. Submission of true and accurate information is a requirement for eligibility and Genentech reserves the right to disqualify patients who do not comply with Genentech Program Terms and Conditions. Genentech reserves the right to rescind, revoke or amend the Program without notice at any time.
Additional terms and conditions apply. Please visit the Co-pay Program website for the full list of Terms and Conditions.
An independent co-pay assistance foundation is a charitable organization providing financial assistance to patients with specific disease states, regardless of treatment. Patients who are commercially or publicly insured, including those covered by Medicare and Medicaid, can contact the foundations directly to request assistance. Eligibility requirements, all aspects of the application process, turnaround times and the type or amount of assistance available (if any) can vary by foundation.
These foundations may be able to help. Please check their websites for up-to-date information.
Advise your patient that these organizations are independent of Genentech and may require the patient to provide personal or financial information directly to the organization to enroll in their respective programs. Genentech cannot share any information the patient has provided to us.
Independent co-pay assistance foundations have their own rules for eligibility. We have no involvement or influence in independent foundation decision-making or eligibility criteria and do not know if a foundation will be able to help your patient. We can only refer your patient to a foundation that supports their disease state. This information is provided as a resource for you. We do not endorse or show preference for any particular foundation. The foundations in this list may not be the only ones that might be able to help your patient.
Genentech Patient Foundation
If patients don’t have health insurance coverage for Evrysdi or have financial concerns and meet eligibility criteria, this program may help:
The Genentech Patient Foundation gives free Evrysdi to people who have been prescribed this medicine and don’t have insurance or that have financial concerns and meet certain eligibility criteria.
Your patient may be eligible if their insurance coverage and income match one of these situations:
- Uninsured patients with incomes under $150,000
- Insured patients without coverage for Evrysdi with incomes under $150,000
- Insured patients with coverage for a Genentech medicine:
- With an out-of-pocket maximum set by their health insurance plan that exceeds 7.5% of their household income
- With household size and income within certain guidelines
For any of these situations, add $25,000 for each extra person in households larger than 4 people.
We encourage insured patients to try other financial assistance options before applying for help from the Genentech Patient Foundation, if possible.
Enrollment Process for the Genentech Patient Foundation
Get started with enrollment by following the steps below.
Option 1: Submit online
If your practice has a registered account for My Patient Solutions, you can get started by logging into your account.
Don't have an account?
Your patient is required to complete the Patient Consent Form. You can either upload their Patient Consent Form as part of your application or have your patient submit the form via fax, text or e-submit.
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An online tool to help you enroll patients in MySMA Support™ and manage your service requests at your convenience.
Option 2: Print & fax or text
Step 1: Print one of the Patient Consent Forms below for your patient to complete.
Step 2: Print and complete the Prescriber Foundation Form below.
Step 3: Submit the completed forms via fax or text.
- Send via fax to 833-999-4363
- Text a photo of the form to 650-877-1111
Both forms are required. We must have both the Patient Consent Form and the Prescriber Foundation Form before we can help you.
What to expect next:
- The request will be processed within five business days upon receipt of both required forms.
- Your office will be contacted to discuss any next steps.
If you have any questions about the criteria, please contact a Foundation Specialist at 888-941-3331 (Monday - Friday, 9 AM - 8 PM ET).
Use our financial assistance tool to see which programs may be right for your patient.
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Commercial insurance: An insurance plan you get from a private health insurance company. This can be insurance from your job, from a plan you bought yourself or from a Health Insurance Marketplace. Medicare and Medicaid are not considered commercial insurance.
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Public insurance: A health insurance plan you get from the federal or state government. This includes Medicare, Medicaid, TRICARE and DoD/VA insurance.
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For example, a household size of 1 with income of less than $75,000 may meet the criteria for assistance. Add $25,000 for each additional person in the household. There is no maximum number of people you may add.
Terms and Conditions
The Product and Administration Co-pay Programs (“Programs”) are valid ONLY for patients with commercial (private or non-governmental) insurance who have a valid prescription for a Food and Drug Administration (FDA)-approved indication of a Genentech medicine. The Product and Administration Co-pay Programs are not available to patients whose prescriptions are reimbursed under any federal, state, or government-funded insurance programs (included by not limited to Medicare, Medicare Advantage, Medigap, Medicaid, TRICARE, Department of Defense, or Veterans Affairs Programs) or where prohibited by law or by the patient's health insurance provider. If at any time a patient begins receiving prescription drug coverage under any such federal, state or government-funded healthcare programs, the patient will no longer be eligible for the Programs. The Programs are not valid if the costs are eligible to be reimbursed in their entirety by private insurance plans or other programs.
Under the Programs, the patient may be required to pay a co-pay. The final amount owed by a patient may be as little as $0 for the Genentech medicine or administration of the Genentech medicine (see Program specific details available at the Program Website). The total patient out-of-pocket cost is dependent on the patient’s health insurance plan. The Programs assist with the cost of the Genentech medicine and the administration of the Genentech medicine only. It does not assist with the cost of other administrations, medicines, procedures or office visit fees. After reaching the maximum Programs’ benefit amounts, the patient will be responsible for all remaining out-of-pocket expenses. The Programs’ benefit amounts cannot exceed the patient’s out-of-pocket expenses for the Genentech medicine or administration fees of the Genentech medicine. The maximum Programs’ benefits will reset every January 1st. The Programs are not health insurance or a benefit plan. The patient’s non-governmental insurance is the primary payer. The Programs do not obligate use of any specific medicine or provider. The Drug Co-pay Program is not available or valid for patients receiving free medicine from the Genentech Patient Foundation. The Administration Co-pay Program is valid for patients receiving free medicine from the Genentech Patient Foundation. The Product and Administration Programs are not valid for patients receiving assistance from any other charitable organization for the same expenses covered by the Programs. The Programs’ benefits cannot be combined with any other rebate, free trial or other offer for the Genentech medicine or administration of the Genentech medicine. No party may seek reimbursement for all or any part of the benefits received through these Programs.
The Programs may be accepted by participating pharmacies, physicians’ offices or hospitals. Once a patient is enrolled, the Programs will honor claims with a date of service that precedes the Programs’ enrollment by 180 days. Claims must be submitted within 365 days from the date of service unless otherwise indicated. Use of these Programs must be consistent with all relevant health insurance requirements. Participating patients, pharmacies, physicians’ offices and hospitals are responsible for reporting the receipt of all the Programs’ benefits as required by any insurer or by law. Programs’ benefits may not be sold, purchased, traded or offered for sale.
The patient or their guardian must be 18 years of age or older to receive assistance from the Programs. The Programs are only valid in the United States and U.S. Territories and are void where prohibited by law. The Drug Co-pay Program shall follow state restrictions in relation to AB-rated generic equivalents (e.g., MA, CA) where applicable. The Administration Co-pay Program is not valid for patients who reside or receive treatment in a restricted state (e.g. Massachusetts or Rhode Island). Eligible patients will be automatically re-enrolled in the Programs on an annual basis. Eligible patients will be removed from the Programs after 3 years of inactivity (e.g., no claims submitted in a 3-year timeframe). Patients who choose reimbursement via virtual debit card will have access to the patient’s funds as long as the patient's virtual debit card is valid and the patient is active in the Programs. Once a patient’s virtual debit card has expired and they are no longer active in the program, the funds will be removed from the virtual debit card. Programs’ eligibility and automatic re-enrollment are contingent upon the patient’s ability to meet all the requirements set forth by the Programs. Healthcare providers may not advertise or otherwise use the Programs as a means of promoting their services or Genentech medicines to patients.
The value of the Programs is intended exclusively for the benefit of the patient. The funds made available through the Programs may only be used to reduce the out-of-pocket costs for the patient enrolled in the Programs. The Programs are not intended for the benefit of third parties, including without limitation third party payers, pharmacy benefit managers, or their agents. If Genentech determines that a third party has implemented programs that adjust patient cost-sharing obligations based on the availability of support under the Programs and/or excludes the assistance provided under the Programs from counting towards the patient’s deductible or out-of-pocket cost limitations, Genentech may impose a per fill cap on the cost-sharing assistance available under the Programs. Submission of true and accurate information is a requirement for eligibility and Genentech reserves the right to disqualify patients who do not comply with Genentech Program Terms and Conditions. Genentech reserves the right to rescind, revoke or amend the Program without notice at any time.
Terms and Conditions
The Co-pay Program (“Program”) is valid ONLY for patients with commercial (private or non-governmental) insurance who have a valid prescription for a Food and Drug Administration (FDA)-approved indication of a Genentech medicine. The Program is not available to patients whose prescriptions are reimbursed under any federal, state, or government-funded insurance programs (included but not limited to Medicare, Medicare Advantage, Medigap, Medicaid, TRICARE, Department of Defense, or Veterans Affairs Programs) or where prohibited by law or by the patient's health insurance provider. If at any time a patient begins receiving prescription drug coverage under any such federal, state or government-funded healthcare programs, the patient will no longer be eligible for the Program. The Program is not valid for Genentech medicines that are eligible to be reimbursed in their entirety by private insurance plans or other programs.
Under the Program, the patient may be required to pay a co-pay. The final amount owed by a patient may be as little as $0 for the Genentech medicine (see Program specific details available on the Program Website). The total patient out-of-pocket cost is dependent on the patient’s health insurance plan. The Program assists with the cost of the Genentech medicine only. It does not assist with the cost of other medicines, procedures or office visit fees. After reaching the maximum annual Program benefit amount, the patient will be responsible for all remaining out-of-pocket expenses. The Program benefit amount cannot exceed the patient’s out-of-pocket expenses for the Genentech medicine. The maximum Program benefit will reset every January 1st. The Program is not health insurance or a benefit plan. The patient’s non-governmental insurance is the primary payer. The Program does not obligate the use of any specific medicine or provider. Patients receiving assistance from charitable free medicine programs (such as the Genentech Patient Foundation) or any other charitable organizations for the same expenses covered by the Program are not eligible. The Program benefit cannot be combined with any other rebate, free trial or other offer for the Genentech medicine. No party may seek reimbursement for all or any part of the benefit received through the Program.
The Program may be accepted by participating pharmacies, physicians’ offices or hospitals. Once a patient is enrolled, the Program will honor claims with a date of service that precedes the Program enrollment date up to 180 days. Claims must be submitted within 365 days from the date of service unless otherwise indicated. Use of the Program must be consistent with all relevant health insurance requirements. Participating patients, pharmacies, physicians’ offices and hospitals are responsible for reporting the receipt of all Program benefits as required by any insurer or by law. Programs’ benefits may not be sold, purchased, traded or offered for sale.
The patient or their guardian must be 18 years of age or older to receive Program assistance. The Program is only valid in the United States and U.S. Territories, is void where prohibited by law and shall follow state restrictions in relation to AB-rated generic equivalents (e.g., MA, CA) where applicable. Eligible patients will be automatically re-enrolled in the Program on an annual basis each January 1st. Eligible patients will be removed from the Program after 3 years of inactivity (e.g., no claims submitted in a 3-year timeframe). Patients who choose reimbursement via virtual debit card will have access to the patient’s funds as long as the patient's virtual debit card is valid and the patient is active in the Program. Once a patient's virtual debit card has expired and they are no longer active in the program, the funds will be removed from the virtual debit card. Program eligibility and automatic re-enrollment are contingent upon the patient’s ability to meet all requirements set forth by the Program. Healthcare providers may not advertise or otherwise use the Program as a means of promoting their services or Genentech medicines to patients.
The value of the Program is intended exclusively for the benefit of the patient. The funds made available through the Program may only be used to reduce the out-of-pocket costs for the patient enrolled in the Program. The Program is not intended for the benefit of third parties, including without limitation third party payers, pharmacy benefit managers, or their agents. If Genentech determines that a third party has implemented a program that adjusts patient cost-sharing obligations based on the availability of support under the Program and/or excludes the assistance provided under the Program from counting towards the patient’s deductible or out-of-pocket cost limitations, Genentech may impose a per fill cap on the cost-sharing assistance available under the Program. Submission of true and accurate information is a requirement for eligibility and Genentech reserves the right to disqualify patients who do not comply with Genentech Program Terms and Conditions. Genentech reserves the right to rescind, revoke or amend the Program without notice at any time.
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Evrysdi® (risdiplam) Prescribing Information. Genentech, Inc.
Evrysdi® (risdiplam) Prescribing Information. Genentech, Inc.
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Poirier A, Weetall M, Heinig K, et al. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol Res Perspect. 2018;6(6):1-12.
Poirier A, Weetall M, Heinig K, et al. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol Res Perspect. 2018;6(6):1-12.
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Farrar MA, Park SB, Vucic S, et al. Emerging therapies and challenges in spinal muscular atrophy. Ann Neurol. 2017;81(3):355-368.
Farrar MA, Park SB, Vucic S, et al. Emerging therapies and challenges in spinal muscular atrophy. Ann Neurol. 2017;81(3):355-368.
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Chiriboga CA, Bruno C, Duong T, et al. JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam. J Neurol. 2024;271(8):4871-4884.
Chiriboga CA, Bruno C, Duong T, et al. JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam. J Neurol. 2024;271(8):4871-4884.
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Data on file. Genentech USA, Inc.
Data on file. Genentech USA, Inc.
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Shababi M, Lorson CL, Rudnik‐Schöneborn SS. Spinal muscular atrophy: a motor neuron disorder or a multi‐organ disease? J Anat. 2014;224(1):15-28.
Shababi M, Lorson CL, Rudnik‐Schöneborn SS. Spinal muscular atrophy: a motor neuron disorder or a multi‐organ disease? J Anat. 2014;224(1):15-28.
-
Aslesh T, Yokota T. Restoring SMN Expression: An overview of the therapeutic developments for the treatment of spinal muscular atrophy. Cells. 2022;11(3):417.
Aslesh T, Yokota T. Restoring SMN Expression: An overview of the therapeutic developments for the treatment of spinal muscular atrophy. Cells. 2022;11(3):417.
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Ojala KS, Reedich EJ, DiDonato CJ, Meriney SD. In search of a cure: the development of therapeutics to alter the progression of spinal muscular atrophy. Brain Sci. 2021;11(2):194.
Ojala KS, Reedich EJ, DiDonato CJ, Meriney SD. In search of a cure: the development of therapeutics to alter the progression of spinal muscular atrophy. Brain Sci. 2021;11(2):194.
-
Cances C, Vlodavets D, Comi GP, et al; on behalf of ANCHOVY Working Group. Natural history of type 1 spinal muscular atrophy: a retrospective, global, multicenter study. Orphanet J Rare Dis. 2022;17(1):300.
Cances C, Vlodavets D, Comi GP, et al; on behalf of ANCHOVY Working Group. Natural history of type 1 spinal muscular atrophy: a retrospective, global, multicenter study. Orphanet J Rare Dis. 2022;17(1):300.
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Yeo CJJ, Darras BT. Overturning the paradigm of spinal muscular atrophy as just a motor neuron disease. Pediatr Neurol. 2020;109:12-19.
Yeo CJJ, Darras BT. Overturning the paradigm of spinal muscular atrophy as just a motor neuron disease. Pediatr Neurol. 2020;109:12-19.
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Lipnick SL, Agniel DM, Aggarwal R, et al. Systemic nature of spinal muscular atrophy revealed by studying insurance claims. PLoS One. 2019;14(3):e0213680.
Lipnick SL, Agniel DM, Aggarwal R, et al. Systemic nature of spinal muscular atrophy revealed by studying insurance claims. PLoS One. 2019;14(3):e0213680.
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Mercuri E, Deconinck N, Mazzone ES, et al; on behalf of the SUNFISH Study Group. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH Part 2): A phase 3, double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2022;21(1):42-52.
Mercuri E, Deconinck N, Mazzone ES, et al; on behalf of the SUNFISH Study Group. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH Part 2): A phase 3, double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2022;21(1):42-52.
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Finkel RS, Farrar MA, Servais L, et al. RAINBOWFISH: Primary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA). Presented at: Congress of the World Muscle Society (WMS); October 3-7, 2023; Charleston, SC.
Finkel RS, Farrar MA, Servais L, et al. RAINBOWFISH: Primary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA). Presented at: Congress of the World Muscle Society (WMS); October 3-7, 2023; Charleston, SC.
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Cure SMA State of SMA 2023 Report. Cure SMA. Accessed May 13, 2025. https://www.curesma.org/wp-content/uploads/2024/06/9042024_State-of-SMA_vWeb.pdf
Cure SMA State of SMA 2023 Report. Cure SMA. Accessed May 13, 2025. https://www.curesma.org/wp-content/uploads/2024/06/9042024_State-of-SMA_vWeb.pdf
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Wan HWY, Carey KA, D’Silva A, Kasparian NA, Farrar MA. “Getting ready for the adult world”: how adults with spinal muscular atrophy perceive and experience healthcare, transition and well-being. Orphanet J Rare Dis. 2019;14(1):74.
Wan HWY, Carey KA, D’Silva A, Kasparian NA, Farrar MA. “Getting ready for the adult world”: how adults with spinal muscular atrophy perceive and experience healthcare, transition and well-being. Orphanet J Rare Dis. 2019;14(1):74.
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Sumner CJ, Crawford TO. Two breakthrough gene-targeted treatments for spinal muscular atrophy: challenges remain. J Clin Invest. 2018;128(8):3219-3227.
Sumner CJ, Crawford TO. Two breakthrough gene-targeted treatments for spinal muscular atrophy: challenges remain. J Clin Invest. 2018;128(8):3219-3227.
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Voice of the patient report: spinal muscular atrophy (SMA). Cure SMA. Accessed May 13, 2025. https://curesma.wpengine.com/wp-content/uploads/2018/01/SMA-VoP-for-publication-1-22-2018.pdf
Voice of the patient report: spinal muscular atrophy (SMA). Cure SMA. Accessed May 13, 2025. https://curesma.wpengine.com/wp-content/uploads/2018/01/SMA-VoP-for-publication-1-22-2018.pdf
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Aponte Ribero V, Martí Y, Batson S, et al. Systematic literature review of the natural history of spinal muscular atrophy: motor function, scoliosis, and contractures. Neurology. 2023;101(21):e2103-e2113.
Aponte Ribero V, Martí Y, Batson S, et al. Systematic literature review of the natural history of spinal muscular atrophy: motor function, scoliosis, and contractures. Neurology. 2023;101(21):e2103-e2113.
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Schroth M, Deans J, Arya K, et al. Spinal muscular atrophy update in best practices: recommendations for diagnosis considerations. Neurol Clin Pract. 2024;14(4).
Schroth M, Deans J, Arya K, et al. Spinal muscular atrophy update in best practices: recommendations for diagnosis considerations. Neurol Clin Pract. 2024;14(4).
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Singh RN, Ottesen EW, Singh NN. The first orally deliverable small molecule for the treatment of spinal muscular atrophy. Neurosci Insights. 2020;15:1-11.
Singh RN, Ottesen EW, Singh NN. The first orally deliverable small molecule for the treatment of spinal muscular atrophy. Neurosci Insights. 2020;15:1-11.
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FDA approves Genentech’s Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older. News Release. Genentech USA, Inc.; August 7, 2020. Accessed May 13, 2025. https://www.gene.com/media/press-releases/14866/2020-08-07/fda-approves-genentechs-evrysdi-risdiplam
FDA approves Genentech’s Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older. News Release. Genentech USA, Inc.; August 7, 2020. Accessed May 13, 2025. https://www.gene.com/media/press-releases/14866/2020-08-07/fda-approves-genentechs-evrysdi-risdiplam
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Baranello G, Boespflug-Tanguy O, Darras BT, et al. FIREFISH Part 1: 24-month safety and exploratory outcomes of risdiplam (RG7916) in infants with type 1 spinal muscular atrophy (SMA). Supplement. Presented at: International Annual Congress of the World Muscle Society; September 28-October 2, 2020; Virtual.
Baranello G, Boespflug-Tanguy O, Darras BT, et al. FIREFISH Part 1: 24-month safety and exploratory outcomes of risdiplam (RG7916) in infants with type 1 spinal muscular atrophy (SMA). Supplement. Presented at: International Annual Congress of the World Muscle Society; September 28-October 2, 2020; Virtual.
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Day JW, Annoussamy M, Baranello G, et al. SUNFISH Part 1: 24-month safety and exploratory outcomes of risdiplam (RG7916) treatment in patients with type 2 or 3 spinal muscular atrophy (SMA). Presented at: 2020 Virtual SMA Conference; June 8-12, 2020; Virtual.
Day JW, Annoussamy M, Baranello G, et al. SUNFISH Part 1: 24-month safety and exploratory outcomes of risdiplam (RG7916) treatment in patients with type 2 or 3 spinal muscular atrophy (SMA). Presented at: 2020 Virtual SMA Conference; June 8-12, 2020; Virtual.
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Kletzl H, Cleary Y, Grimsey P, Gerber M, Scalco RS. Risdiplam: pharmacokinetic, pharmacodynamic, safety and efficacy exposure response analyses. Poster presented at: Cure SMA 2022 Research and Clinical Care Meeting; June 15-17, 2022; Anaheim, CA.
Kletzl H, Cleary Y, Grimsey P, Gerber M, Scalco RS. Risdiplam: pharmacokinetic, pharmacodynamic, safety and efficacy exposure response analyses. Poster presented at: Cure SMA 2022 Research and Clinical Care Meeting; June 15-17, 2022; Anaheim, CA.
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Baranello G, Darras BT, Day JW, et al. Risdiplam in type 1 spinal muscular atrophy. N Engl J Med. 2021;384(10)(suppl 1):915-923.
Baranello G, Darras BT, Day JW, et al. Risdiplam in type 1 spinal muscular atrophy. N Engl J Med. 2021;384(10)(suppl 1):915-923.
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Mercuri E, Deconinck N, Mazzone ES, et al; on behalf of the SUNFISH Study Group. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH Part 2): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2021;21(1)(suppl 1):42-52.
Mercuri E, Deconinck N, Mazzone ES, et al; on behalf of the SUNFISH Study Group. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH Part 2): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2021;21(1)(suppl 1):42-52.
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Mercuri E, Baranello G, Boespflug-Tanguy O, et al. Risdiplam in types 2 and 3 spinal muscular atrophy: a randomised, placebo-controlled, dose-finding trial followed by 24 months of treatment. Eur J Neurol. 2022;30(7):1945-1956.
Mercuri E, Baranello G, Boespflug-Tanguy O, et al. Risdiplam in types 2 and 3 spinal muscular atrophy: a randomised, placebo-controlled, dose-finding trial followed by 24 months of treatment. Eur J Neurol. 2022;30(7):1945-1956.
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Darras BT, Boespflug-Tanguy O, Day JW, et al; on behalf of the FIREFISH Working Group. FIREFISH Parts 1 and 2: 24-month safety and efficacy of risdiplam in infants with type 1 SMA. Presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 13-16, 2022; Nashville, TN.
Darras BT, Boespflug-Tanguy O, Day JW, et al; on behalf of the FIREFISH Working Group. FIREFISH Parts 1 and 2: 24-month safety and efficacy of risdiplam in infants with type 1 SMA. Presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 13-16, 2022; Nashville, TN.
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PTC Therapeutics announces FDA approval of Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy in adults and children 2 months and older. News Release. PTC Therapeutics. August 7, 2020. Accessed May 13, 2025. https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-fda-approval-evrysditm-risdiplam
PTC Therapeutics announces FDA approval of Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy in adults and children 2 months and older. News Release. PTC Therapeutics. August 7, 2020. Accessed May 13, 2025. https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-fda-approval-evrysditm-risdiplam
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Bérard C, Payan C, Hodgkinson I, Fermanian J; on behalf of the MFM Collaborative Study Group. A motor function measure scale for neuromuscular diseases. Construction and validation study. Neuromuscul Disord. 2005;15(7):463-470.
Bérard C, Payan C, Hodgkinson I, Fermanian J; on behalf of the MFM Collaborative Study Group. A motor function measure scale for neuromuscular diseases. Construction and validation study. Neuromuscul Disord. 2005;15(7):463-470.
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de Lattre C, Payan C, Vuillerot C, et al. Motor function measure: validation of a short form for young children with neuromuscular diseases. Arch Phys Med Rehabil. 2013;94(11):2218-2226.
de Lattre C, Payan C, Vuillerot C, et al. Motor function measure: validation of a short form for young children with neuromuscular diseases. Arch Phys Med Rehabil. 2013;94(11):2218-2226.
-
Vuillerot C, Payan C, Iwaz J, Ecochard R, Bérard C; MFM Spinal Muscular Atrophy Study Group. Responsiveness of the motor function measure in patients with spinal muscular atrophy. Arch Phys Med Rehabil. 2013;94(8):1555-1561.
Vuillerot C, Payan C, Iwaz J, Ecochard R, Bérard C; MFM Spinal Muscular Atrophy Study Group. Responsiveness of the motor function measure in patients with spinal muscular atrophy. Arch Phys Med Rehabil. 2013;94(8):1555-1561.
-
Duong T, Staunton H, Braid J, et al. A patient-centered evaluation of meaningful change on the 32-item motor function measure in spinal muscular atrophy using qualitative and quantitative data. Front Neurol. 2022;12:770423.
Duong T, Staunton H, Braid J, et al. A patient-centered evaluation of meaningful change on the 32-item motor function measure in spinal muscular atrophy using qualitative and quantitative data. Front Neurol. 2022;12:770423.
-
Servais L, Oskoui M, Day JW, et al; on behalf of the SUNFISH Study Group. SUNFISH Parts 1 and 2: 4-year efficacy and safety of risdiplam in types 2 and 3 spinal muscular atrophy (SMA). Presented at: American Academy of Neurology; April 22-27, 2023; Boston, MA.
Servais L, Oskoui M, Day JW, et al; on behalf of the SUNFISH Study Group. SUNFISH Parts 1 and 2: 4-year efficacy and safety of risdiplam in types 2 and 3 spinal muscular atrophy (SMA). Presented at: American Academy of Neurology; April 22-27, 2023; Boston, MA.
-
Mazzone ES, Mayhew A, Montes J, et al. Revised upper limb module for spinal muscular atrophy: Development of a new module. Muscle Nerve. 2017;55(6):869-874.
Mazzone ES, Mayhew A, Montes J, et al. Revised upper limb module for spinal muscular atrophy: Development of a new module. Muscle Nerve. 2017;55(6):869-874.
-
Pera MC, Coratti G, Mazzone ES, et al. Revised upper limb module for spinal muscular atrophy: 12 month changes. Muscle Nerve. 2019;59(4):426-430.
Pera MC, Coratti G, Mazzone ES, et al. Revised upper limb module for spinal muscular atrophy: 12 month changes. Muscle Nerve. 2019;59(4):426-430.
-
Day JW, Deconinck N, Mazzone E, et al. SUNFISH Part 2: 24-month efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA). Poster presented at: Cure SMA Virtual Research & Clinical Care Meeting; June 9–11, 2021; Virtual.
Day JW, Deconinck N, Mazzone E, et al. SUNFISH Part 2: 24-month efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA). Poster presented at: Cure SMA Virtual Research & Clinical Care Meeting; June 9–11, 2021; Virtual.
-
Baranello G, Darras BT, Day JW, et al. Risdiplam in type 1 spinal muscular atrophy. N Engl J Med. 2021;384:915-923.
Baranello G, Darras BT, Day JW, et al. Risdiplam in type 1 spinal muscular atrophy. N Engl J Med. 2021;384:915-923.
-
Mazurkiewicz-Bełdzińska M, Baranello G, Boespflug-Tanguy O, et al; on behalf of the FIREFISH Study Group. FIREFISH Parts 1 and 2: 5-year efficacy and safety of risdiplam in type 1 SMA. Roche USA, Inc.; 2024. Presented at: Cure SMA; June 6-9, 2024; Austin, Texas.
Mazurkiewicz-Bełdzińska M, Baranello G, Boespflug-Tanguy O, et al; on behalf of the FIREFISH Study Group. FIREFISH Parts 1 and 2: 5-year efficacy and safety of risdiplam in type 1 SMA. Roche USA, Inc.; 2024. Presented at: Cure SMA; June 6-9, 2024; Austin, Texas.
-
Hammersmith Infant Neurological Examination. UNM Health. Accessed May 13, 2025. https://unmhealth.org/services/development-disabilities/programs/other-disability-programs/cerebral-palsy-taskforce/_media/hammersmith-infant-neurological-exam.pdf.
Hammersmith Infant Neurological Examination. UNM Health. Accessed May 13, 2025. https://unmhealth.org/services/development-disabilities/programs/other-disability-programs/cerebral-palsy-taskforce/_media/hammersmith-infant-neurological-exam.pdf.
-
Prior TW, Leach ME, Finanger EL. Spinal Muscular Atrophy. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Amemiya A, eds. GeneReviews®. Seattle (WA): University of Washington, Seattle; February 24, 2000.
Prior TW, Leach ME, Finanger EL. Spinal Muscular Atrophy. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Amemiya A, eds. GeneReviews®. Seattle (WA): University of Washington, Seattle; February 24, 2000.
-
Servais L, Finkel RS, Vlodavets D, et al; on behalf of the RAINBOWFISH Study Group. RAINBOWFISH: 2-year efficacy and safety data of risdiplam in infants with presymptomatic SMA. Presented at: Congress of the World Muscle Society (WMS), October 12, 2024; Prague, Czechia
Servais L, Finkel RS, Vlodavets D, et al; on behalf of the RAINBOWFISH Study Group. RAINBOWFISH: 2-year efficacy and safety data of risdiplam in infants with presymptomatic SMA. Presented at: Congress of the World Muscle Society (WMS), October 12, 2024; Prague, Czechia
-
An open-label study of risdiplam in infants with genetically diagnosed and presymptomatic spinal muscular atrophy. Clinicaltrials.gov. Accessed May 13, 2025. https://clinicaltrials.gov/study/NCT03779334
An open-label study of risdiplam in infants with genetically diagnosed and presymptomatic spinal muscular atrophy. Clinicaltrials.gov. Accessed May 13, 2025. https://clinicaltrials.gov/study/NCT03779334
-
De Vivo DC, Bertini E, Swoboda KJ, et al. Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study. Neuromuscul Disord. 2019;29(11):842-856.
De Vivo DC, Bertini E, Swoboda KJ, et al. Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study. Neuromuscul Disord. 2019;29(11):842-856.
-
Strauss KA, Farrar MA, Muntoni F, et al. Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial. Nat Med. 2022;28(7):1381-1389.
Strauss KA, Farrar MA, Muntoni F, et al. Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial. Nat Med. 2022;28(7):1381-1389.
-
Chiriboga CA, Bruno C, Duong T, et al; on behalf of the JEWELFISH Study Group. Risdiplam in patients previously treated with other therapies for spinal muscular atrophy: An interim analysis from the JEWELFISH study. Neurol Ther. 2023;12(2):543-557.
Chiriboga CA, Bruno C, Duong T, et al; on behalf of the JEWELFISH Study Group. Risdiplam in patients previously treated with other therapies for spinal muscular atrophy: An interim analysis from the JEWELFISH study. Neurol Ther. 2023;12(2):543-557.
-
Oskoui M, Day JW, Deconinck N, et al. SUNFISH Parts 1 and 2: 4-year efficacy and safety of risdiplam in types 2 and 3 spinal muscular atrophy (SMA). Presented at: Muscular Dystrophy Association (MDA) Clinical and Scientific Conference; March 19-22, 2023; Dallas, TX.
Oskoui M, Day JW, Deconinck N, et al. SUNFISH Parts 1 and 2: 4-year efficacy and safety of risdiplam in types 2 and 3 spinal muscular atrophy (SMA). Presented at: Muscular Dystrophy Association (MDA) Clinical and Scientific Conference; March 19-22, 2023; Dallas, TX.
-
IND Application Reporting: Safety Reports. U.S. Food & Drug. Accessed May 13, 2025. https://www.fda.gov/drugs/investigational-new-drug-ind-application/ind-application-reporting-safety-reports
IND Application Reporting: Safety Reports. U.S. Food & Drug. Accessed May 13, 2025. https://www.fda.gov/drugs/investigational-new-drug-ind-application/ind-application-reporting-safety-reports
-
Nascimento A, Day JW, Deconinck N, et al; on behalf of the SUNFISH Working Group. SUNFISH Part 2: 24-month efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA). Presented at: Congress of the World Muscle Society (WMS); September 20-24, 2021; Virtual.
Nascimento A, Day JW, Deconinck N, et al; on behalf of the SUNFISH Working Group. SUNFISH Part 2: 24-month efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA). Presented at: Congress of the World Muscle Society (WMS); September 20-24, 2021; Virtual.
-
Kletzl H, Heinig K, Jaber B, et al. Bioequivalence and food effect assessment for a room-temperature stable risdiplam tablet formulation in healthy volunteers. Presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 3-6, 2024; Orlando, FL.
Kletzl H, Heinig K, Jaber B, et al. Bioequivalence and food effect assessment for a room-temperature stable risdiplam tablet formulation in healthy volunteers. Presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 3-6, 2024; Orlando, FL.
-
Evrysdi® (risdiplam) Instructions For Use. Genentech, Inc.
Evrysdi® (risdiplam) Instructions For Use. Genentech, Inc.
-
Coşkun MB, Yalçın İ, Özarslan C. Physical properties of sweet corn seed (Zea mays saccharata sturt.). J Food Eng. 2006;74(4):523-528.
Coşkun MB, Yalçın İ, Özarslan C. Physical properties of sweet corn seed (Zea mays saccharata sturt.). J Food Eng. 2006;74(4):523-528.
-
Pharmaceutical issues when crushing, opening or Splitting Oral Dosage Forms. Royal Pharmaceutical Society. Accessed May 13, 2025. https://www.rpharms.com/Portals/0/RPS%20document%20library/Open%20access/Support/toolkit/pharmaceuticalissuesdosageforms-(2).pdf.
Pharmaceutical issues when crushing, opening or Splitting Oral Dosage Forms. Royal Pharmaceutical Society. Accessed May 13, 2025. https://www.rpharms.com/Portals/0/RPS%20document%20library/Open%20access/Support/toolkit/pharmaceuticalissuesdosageforms-(2).pdf.
-
Evrysdi® (risdiplam) Instructions For Constitution. Genentech, Inc.
Evrysdi® (risdiplam) Instructions For Constitution. Genentech, Inc.
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