For Patients and Caregivers

Access and reimbursement resources for your practice

MySMA Support™ offers a range of access and reimbursement resources for your patients and practice after Evrysdi is prescribed, including help with benefits investigations (BIs), resources for prior authorizations (PAs), sample billing and coding information, resources for denials and appeals, information about distribution and referrals to potential financial assistance options.

Quick Links

Help with Coverage (BIs and PAs)
Reimbursement (Billing, Coding and Appeals)
Online Patient Enrollment
Evrysdi Distribution

Coverage

Get help understanding insurance benefits and coverage, such as with benefits investigations and prior authorization resources.

Benefits Investigations

MySMA Support™ can conduct a benefits investigation (BI) which can determine:

  • If treatment is covered
  • If treatment is denied
  • If a prior authorization or pre-determination is required*
  • If your patient's insurance plan has a mandated or preferred specialty pharmacy

*If your patient’s request for a prior authorization is not granted, your MySMA Support™ specialist can work with you to determine your next steps.

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.

  • 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 the Evrysdi Start Form below and have your patient complete the patient consent section of this form.

Step 2: Print and complete the Prescriber Service Form below.

Step 2: Submit the completed forms via fax or text.

Both sections of the form are required. We must have both sections of the completed Evrysdi Start 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.

The completion and submission of coverage- or reimbursement-related documentation are the responsibility of the patient and healthcare provider. Genentech makes no representation or guarantee concerning coverage or reimbursement for any service or item.

Case Managers

Evrysdi Case Managers

A Case Manager may assist during your patient's treatment with access and reimbursement.

Learn more about MySMA Support

Evrysdi Starter Program

Patients facing a coverage delay may be eligible for the Evrysdi Starter Program while awaiting a coverage determination. If you would like your patient considered for the Evrysdi Starter Program, you can indicate this when enrolling in MySMA Support with the Evrysdi Start Form.

Eligible patients can receive up to a ~30-day supply of Evrysdi. If the patient continues to experience a coverage delay, the patient may be eligible for one refill (up to a ~30-day supply) of Evrysdi. Once coverage has been determined, the patient no longer qualifies for the Evrysdi Starter Program.

Subject to eligibility requirements and terms and conditions. This program is void where prohibited by law and may not be used in or by residents of restricted states, if applicable.

View full TERMS AND CONDITIONS

Reimbursement

Sample coding information and resources for denials and appeals

Evrysdi Sample Coding

This coding information may assist you as you complete the payer forms for Evrysdi. These tables are provided for informational purposes only. Please visit CMS.gov or other payers’ websites to obtain additional guidance on their processes related to billing and coding.

Sample Coding: Spinal muscular atrophy (SMA)

Correct coding is the responsibility of the provider submitting the claim for the item or service. Please check with the payer to verify codes and special billing requirements. Genentech does not make any representation or guarantee concerning reimbursement or coverage for any service or item.

Appeals

If your patient’s health insurance plan has issued a denial, your MySMA Support™ specialist can provide resources as you prepare an appeal submission, as per your patient’s plan requirements. 

If a plan issues a denial: 

  1. The denial should be reviewed, along with the health insurance plan’s guidelines to determine what to include in your patient’s appeal submission.
  2. Your MySMA Support™ specialist has local payer coverage expertise and can help you determine specific requirements for your patient.

A sample appeal letter and additional considerations are available on the Practice Forms & Documents page.

Appeals cannot be completed or submitted by Genentech on your behalf.

Online Patient Enrollment

My Patient Solutions is an online tool to help you enroll patients in MySMA Support™ and manage your service requests, all through one portal. It allows you the flexibility to work with MySMA Support™ when it’s convenient for you.

With My Patient Solutions, you can:

  • Enroll and re-enroll patients in MySMA Support™ and/or the Genentech Patient Foundation
  • View Genentech Patient Foundation eligibility and coordinate shipment
  • Communicate with your MySMA Support™ specialist
  • Easily identify next steps for service requests
  • View Benefits Investigation reports for all your enrolled patients
  • Follow up on prior authorizations or appeals

 

How to register

Account registration can be completed by one person for the entire practice and for multiple practice locations. For help with registration or if you have questions, call us at 877-GENENTECH (877-436-3683) (9AM-8PM ET, Monday through Friday).

Register for My Patient Solutions

Evrysdi Distribution

MySMA Support™ works with specialty pharmacies (SPs) to help patients receive their prescribed Genentech medicines.

In addition to distributing medicines, a specialty pharmacy may provide the following services:

  • Reimbursement resources
  • Clinical services to help patients throughout their treatment
  • The ability to manage the specialty handling and shipping needs linked with many specialty therapies

You can work with your preferred specialty pharmacy or contact MySMA Support™ to learn which specialty pharmacy the patient’s health insurance plan mandates or prefers.

A specialty pharmacy (SP)* prepares and ships Evrysdi directly to patients each month. Although the SP is not a part of Genentech, it is an important part of the MySMA Support team.

The SP will call the patient or caregiver to welcome them to the program and to schedule delivery times that work best for them. Remind the patient or caregiver to answer calls from the SP to prevent any delays in treatment.

For a full list of in-network specialty pharmacies, please visit the Genentech Access Solutions website or contact MySMA Support™ at 833-387-9734.

*Specialty pharmacies are not part of Genentech and maintain independence in their operations and in their role as a health care provider.

Genentech does not influence or advocate the use of any one specialty distributor or specialty pharmacy. We make no representation or guarantee of service or coverage of any item. For any product-specific distribution questions, call MySMA Support™ at 833-387-9734 (9AM-8PM ET, Monday through Friday).

Contact Us

Need more help? Contact MySMA Support™

Call 833-387-9734 (Mon.–Fri., 6AM–5PM PST).

Contact Us

Questions? Contact Evrysdi Co-pay Support

Call (833) 387-9734 (Monday–Friday, 9AM–8PM ET, except major holidays)

Financial support

Financial Support

Find the right financial resources option for your patients.

Explore options
NEXT: Enrollment Forms & Documents

  • We are open from 9AM-8PM ET, Monday through Friday, except for the following holidays:

    • New Year’s Day
    • Martin Luther King, Jr. Day
    • Memorial Day
    • Juneteenth
    • Independence Day
    • Labor Day
    • Thanksgiving Holiday (Thursday and Friday)
    • Christmas Day

Important Safety Information and Indication

Indication

EVRYSDI is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

Interactions with Substrates of MATE Transporters

  • Based on in vitro data, Evrysdi may increase plasma concentrations of drugs eliminated via MATE1 or MATE2-K, such as metformin
  • Avoid coadministration of Evrysdi with MATE (multidrug and toxin extrusion) substrates. If coadministration cannot be avoided, monitor for drug-related toxicities and consider dosage reduction of the coadministered drug if needed

Pregnancy & Breastfeeding

  • Evrysdi may cause embryofetal harm when administered to a pregnant woman. In animal studies, administration of Evrysdi during pregnancy and/or lactation resulted in adverse effects on development. Advise pregnant women of the potential risk to the fetus
  • Pregnancy testing is recommended prior to initiating Evrysdi. Advise female patients to use contraception during treatment with Evrysdi and for at least 1 month after the last dose
  • There is a pregnancy exposure registry that monitors pregnancy and fetal/neonatal/infant outcomes in women exposed to Evrysdi during pregnancy. Physicians are encouraged to register patients and pregnant women are encouraged to register themselves by calling 1-833-760-1098 or visiting https://www.evrysdipregnancyregistry.com.
  • The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Evrysdi and any potential adverse effects on the breastfed infant

Potential Effects on Male Fertility

  • Counsel male patients that fertility may be compromised by treatment with Evrysdi. Male patients may consider sperm preservation prior to treatment

Most Common Adverse Reactions

  • The most common adverse reactions in later-onset SMA (incidence in at least 10% of patients treated with Evrysdi and more frequent than control) were fever, diarrhea, and rash
  • The most common adverse reactions in infantile-onset SMA were similar to those observed in later-onset SMA patients. Additionally, adverse reactions with an incidence of at least 10% were upper respiratory tract infection (including nasopharyngitis, rhinitis), lower respiratory tract infection (including pneumonia, bronchitis), constipation, vomiting, and cough
  • The safety profile for presymptomatic patients is consistent with the safety profile for symptomatic SMA patients treated with Evrysdi in clinical trials

You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.

Please see full Prescribing Information for additional Important Safety Information.

    • Evrysdi® (risdiplam) Prescribing Information. Genentech, Inc.

      Evrysdi® (risdiplam) Prescribing Information. Genentech, Inc.

    • 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.

    • 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.

    • 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.

    • Data on file. Genentech USA, Inc.

      Data on file. Genentech USA, Inc.

    • 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.

    • 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.

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      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.

    • 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.

    • 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.

    • 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.

    • 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.

    • 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

    • 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.

    • 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.

    • 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

    • 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.

    • 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).

    • 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.

    • 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

    • 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.

    • 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.

    • 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. 

    • 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.

    • 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.

    • 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.

    • 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.

    • 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.

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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.

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      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.

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      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.

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    • 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

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      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

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