For Patients and Caregivers

NOW APPROVED: Evrysdi tablet formulation1

For illustrative purposes only.

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Please remember: This feature is intended for US healthcare professionals only.


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Please complete the form below if you would like a Genentech Neurological Rare Disease Account Manager to contact you regarding your Evrysdi information request. You should receive a response within 2 business days. Your contact information will not be used for any purpose other than for a representative to respond to your information request.

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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 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.

    • Data on file. Genentech USA, Inc.

      Data on file. Genentech USA, Inc.

    • NIH US National Library of Medicine. Spinal muscular atrophy. https://ghr.nlm.nih.gov/condition/spinal-muscular-atrophy. Accessed June 7, 2021.

      NIH US National Library of Medicine. Spinal muscular atrophy. https://ghr.nlm.nih.gov/condition/spinal-muscular-atrophy. Accessed June 7, 2021.

    • 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.

       

    • 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.

    • Cure SMA. Voice of the patient report: spinal muscular atrophy (SMA). https://curesma.wpengine.com/wp-content/uploads/2018/01/SMA-VoP-for-publication-1-22-2018.pdf. Published January 10, 2018. Accessed June 7, 2021.

      Cure SMA. Voice of the patient report: spinal muscular atrophy (SMA). https://curesma.wpengine.com/wp-content/uploads/2018/01/SMA-VoP-for-publication-1-22-2018.pdf. Published January 10, 2018. Accessed June 7, 2021.

    • Prior TW, Leach ME, Finanger E. Spinal Muscular Atrophy. In: Adam MP, Ardinger HH, Pagon RA, et al., eds. GeneReviews®. Seattle (WA): University of Washington, Seattle; February 24, 2000.

      Prior TW, Leach ME, Finanger E. Spinal Muscular Atrophy. In: Adam MP, Ardinger HH, Pagon RA, et al., eds. GeneReviews®. Seattle (WA): University of Washington, Seattle; February 24, 2000.

    • Hamilton G, Gillingwater TH. Spinal muscular atrophy: going beyond the motor neuron. Trends Mol Med. 2013;19(1):40-50.

      Hamilton G, Gillingwater TH. Spinal muscular atrophy: going beyond the motor neuron. Trends Mol Med. 2013;19(1):40-50.

    • Baranello G, Bloespflug-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). Supplemental presentation at: 2020 Virtual World Muscle Society; September 28-October 2, 2020; Virtual.

      Baranello G, Bloespflug-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). Supplemental presentation at: 2020 Virtual 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). Presentation 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). Presentation at: 2020 Virtual SMA Conference; June 8-12, 2020; Virtual.

    • 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. Poster presented at: Muscular Dystrophy Association Clinical and Scientifi c 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. Poster presented at: Muscular Dystrophy Association Clinical and Scientifi c Conference; March 13-16, 2022; Nashville, TN.

    • Cances C, Vlodavets D, Comi GP, et al; 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; ANCHOVY Working Group. Natural history of type 1 spinal muscular atrophy: a retrospective, global, multicenter study. Orphanet J Rare Dis. 2022;17(1):300.

    • 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.

    • 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. doi: 10.1056/NEJMoa2009965.

      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. doi: 10.1056/NEJMoa2009965.

    • Darras BT, Baranello G, Boespflug-Tanguy O, et al. FIREFISH Part 1: 24-month safety and exploratory outcomes of risdiplam in infants with Type 1 spinal muscular atrophy (SMA). Presentation at: 2021 Virtual MDA Conference; March 15-18, 2021; Virtual.

      Darras BT, Baranello G, Boespflug-Tanguy O, et al. FIREFISH Part 1: 24-month safety and exploratory outcomes of risdiplam in infants with Type 1 spinal muscular atrophy (SMA). Presentation at: 2021 Virtual MDA Conference; March 15-18, 2021; Virtual.

    • 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.

    • Mercuri E, Barisic N, Boespflug-Tanguy O, et al. SUNFISH Part 2: efficacy and safety of risdiplam (RG7916) in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA). Presentation at: 2020 Virtual American Academy of Neurology (AAN) Conference; April 25-May 1, 2020; Virtual.

      Mercuri E, Barisic N, Boespflug-Tanguy O, et al. SUNFISH Part 2: efficacy and safety of risdiplam (RG7916) in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA). Presentation at: 2020 Virtual American Academy of Neurology (AAN) Conference; April 25-May 1, 2020; Virtual.

    • Annoussamy M, Seferian AM, Daron A, et al; NatHis-SMA study group. Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study. Ann Clin Transl Neurol. 2021;8(2):359-373.doi:10.1002/acn3.51281.

      Annoussamy M, Seferian AM, Daron A, et al; NatHis-SMA study group. Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study. Ann Clin Transl Neurol. 2021;8(2):359-373.doi:10.1002/acn3.51281.

    • Bérard C, Payan C, Hodgkinson I, et al., and the MFM Collaborative Study Group. A motor function measure scale for neuromuscular diseases. Construction and validation study. Neuromuscul Disord. 2005;15:463-470.

      Bérard C, Payan C, Hodgkinson I, et al., and the MFM Collaborative Study Group. A motor function measure scale for neuromuscular diseases. Construction and validation study. Neuromuscul Disord. 2005;15:463-470.

    • Trundell D, Le Scouiller S, Le Goff L, et al. Assessment of the validity and reliability of the 32-item Motor Function Measure in individuals with Type 2 or non-ambulant Type 3 spinal muscular atrophy. PLoS One. 2020;15(9):e0238786. doi: 10.1371/journal. pone.0238786.

      Trundell D, Le Scouiller S, Le Goff L, et al. Assessment of the validity and reliability of the 32-item Motor Function Measure in individuals with Type 2 or non-ambulant Type 3 spinal muscular atrophy. PLoS One. 2020;15(9):e0238786. doi: 10.1371/journal. pone.0238786.

    • Vuillerot C, Payan C, Iwaz J, et al. 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, et al. Responsiveness of the motor function measure in patients with spinal muscular atrophy. Arch Phys Med Rehabil. 2013;94(8):1555-1561.

    • Mazzone ES, Mayhew A, Montes J, et al. Revised upper limb module for spinal muscular atrophy: development of a new module. Muscle Nerve. 2017;55:869-874.

      Mazzone ES, Mayhew A, Montes J, et al. Revised upper limb module for spinal muscular atrophy: development of a new module. Muscle Nerve. 2017;55:869-874.

    • Pera MC, Coratti G, Mazzone ES, et al. Revised upper limb module for spinal muscular atrophy: 12 month changes. Muscle Nerve. 2019;59:426-430.

      Pera MC, Coratti G, Mazzone ES, et al. Revised upper limb module for spinal muscular atrophy: 12 month changes. Muscle Nerve. 2019;59:426-430.

    • Mazzella A, Curry M, Belter L, et al. “I have SMA, SMA doesn't have me”: a qualitative snapshot into the challenges, successes, and quality of life of adolescents and young adults with SMA. Orphanet J Rare Dis. 2021;16(1):96.

      Mazzella A, Curry M, Belter L, et al. “I have SMA, SMA doesn't have me”: a qualitative snapshot into the challenges, successes, and quality of life of adolescents and young adults with SMA. Orphanet J Rare Dis. 2021;16(1):96.

    • Wan HWY, Carey KA, D’Silva A, et al. “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, et al. “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.

    • Albers CA, Grieve AJ. Test review: Bayley, N. (2006). Bayley Scales of Infant and Toddler Development–Third Edition. San Antonio, TX: Harcourt Assessment. J Psychoeduc Assess. 2007;25(2):180-198.

      Albers CA, Grieve AJ. Test review: Bayley, N. (2006). Bayley Scales of Infant and Toddler Development–Third Edition. San Antonio, TX: Harcourt Assessment. J Psychoeduc Assess. 2007;25(2):180-198.

    • 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. Poster 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. Poster presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 13-16, 2022; Nashville, TN.

    • Finkel RS, Farrar MA, Vlodavets D, et al, on behalf of the RAINBOWFISH Study Group. RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic SMA. Poster presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 13-16, 2022; Nashville, TN.

      Finkel RS, Farrar MA, Vlodavets D, et al, on behalf of the RAINBOWFISH Study Group. RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic SMA. Poster presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 13-16, 2022; Nashville, TN.

    • Baranello G, Day JW, Deconinck N, et al, on behalf of the SUNFISH Working Group. SUNFISH: efficacy and safety of risdiplam in Types 2 and 3 SMA. Poster presented at: European Paediatric Neurology Society; April 28 - May 2, 2022; Glasgow, UK.

      Baranello G, Day JW, Deconinck N, et al, on behalf of the SUNFISH Working Group. SUNFISH: efficacy and safety of risdiplam in Types 2 and 3 SMA. Poster presented at: European Paediatric Neurology Society; April 28 - May 2, 2022; Glasgow, UK.

    • Chiriboga CA, Bruno C, Duong T, et al. JEWELFISH: 24-month safety, pharmacodynamic and exploratory efficacy data in non-treatment-naïve patients with SMA receiving treatment with risdiplam. Presentation at: 2022 Virtual World Muscle Society (WMS) Conference; October 11-15, 2022; Halifax, Canada.

      Chiriboga CA, Bruno C, Duong T, et al. JEWELFISH: 24-month safety, pharmacodynamic and exploratory efficacy data in non-treatment-naïve patients with SMA receiving treatment with risdiplam. Presentation at: 2022 Virtual World Muscle Society (WMS) Conference; October 11-15, 2022; Halifax, Canada.

    • 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.

    • Iannaccone ST, To TM, Dickendesher T, Shapouri S, Pineda ED. A retrospective analysis of adherence and persistence among risdiplam-treated patients with spinal muscular atrophy (SMA). Poster presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 19-22, 2023; Dallas, TX.

      Iannaccone ST, To TM, Dickendesher T, Shapouri S, Pineda ED. A retrospective analysis of adherence and persistence among risdiplam-treated patients with spinal muscular atrophy (SMA). Poster presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 19-22, 2023; Dallas, TX.

    • Chiriboga CA, Bruno C, Duong T. 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)(suppl 1):4871-4884.

      Chiriboga CA, Bruno C, Duong T. 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)(suppl 1):4871-4884.

    • 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.; October 3, 2020. Accessed October 3, 2024. 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.; October 3, 2020. Accessed October 3, 2024. https://www.gene.com/media/press-releases/14866/2020-08-07/fda-approves-genentechs-evrysdi-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 October 3, 2024. 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 October 3, 2024. https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-fda-approval-evrysditm-risdiplam

    • Spinal Muscular Atrophy. MedlinePlus. Updated September 19, 2024. Accessed October 3, 2024. https://medlineplus.gov/genetics/condition/spinal-muscular-atrophy/.

      Spinal Muscular Atrophy. MedlinePlus. Updated September 19, 2024. Accessed October 3, 2024. https://medlineplus.gov/genetics/condition/spinal-muscular-atrophy/.

    • 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, 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.

    • 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.

    • 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.

    • 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.

    • 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.

    • Trundell D, Le Scouiller S, Staunton H, Gorni K, Vuillerot C. Validity and reliability of the motor function measure (MFM32) in children with neuromuscular disorders (NMDs) and in individuals with type 2 and non-ambulant type 3 spinal muscular atrophy (SMA). Presented at: Cure SMA Researcher Meeting; June 28-July 1, 2019; Anaheim, CA.

      Trundell D, Le Scouiller S, Staunton H, Gorni K, Vuillerot C. Validity and reliability of the motor function measure (MFM32) in children with neuromuscular disorders (NMDs) and in individuals with type 2 and non-ambulant type 3 spinal muscular atrophy (SMA). Presented at: Cure SMA Researcher Meeting; June 28-July 1, 2019; Anaheim, CA.

    • Finkel RS, Farrar MA, Servais L, et al; on behalf of the RAINBOWFISH Study Group. RAINBOWFISH: Primary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA). Presented at: Muscular Dystrophy Association (MDA) Clinical and Scientific Conference; March 3-6, 2024; Orlando, FL.

      Finkel RS, Farrar MA, Servais L, et al; on behalf of the RAINBOWFISH Study Group. RAINBOWFISH: Primary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA). Presented at: Muscular Dystrophy Association (MDA) Clinical and Scientific Conference; March 3-6, 2024; Orlando, FL.

    • Hammersmith Infant Neurological Examination. UBC CPD. Updated July 7, 2017. Accessed October 3, 2024. https://ubccpd.ca/media/1951/download

      Hammersmith Infant Neurological Examination. UBC CPD. Updated July 7, 2017. Accessed October 3, 2024. https://ubccpd.ca/media/1951/download

    • 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 October 3, 2024. 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 October 3, 2024. 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.

    • 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.

    • 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 (MDA) Clinical & 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 (MDA) Clinical & Scientific Conference; March 3-6, 2024; Orlando, FL.

    • Ratni H, Scalco RS, Stephan AH. Risdiplam, the first approved small molecule splicing modifier drug as a blueprint for future transformative medicines. ACS Med Chem Lett. 2021;12(6):874-877.

      Ratni H, Scalco RS, Stephan AH. Risdiplam, the first approved small molecule splicing modifier drug as a blueprint for future transformative medicines. ACS Med Chem Lett. 2021;12(6):874-877.

    • Physical properties of sweet corn seed. ScienceDirect. Accessed October 25, 2024. https://www.sciencedirect.com/science/article/abs/pii/S0260877405001925

      Physical properties of sweet corn seed. ScienceDirect. Accessed October 25, 2024. https://www.sciencedirect.com/science/article/abs/pii/S0260877405001925

    • Pharmaceutical issues when crushing, opening or splitting oral dosage forms. Royal Pharmaceutical Society. June 2011. Accessed January 28, 2025. https://www.rpharms.com/Portals/0/RPS%20document%20library/Open%20access/Support/toolkit/pharmaceuticalissuesdosageforms-%282%29.pdf

      Pharmaceutical issues when crushing, opening or splitting oral dosage forms. Royal Pharmaceutical Society. June 2011. Accessed January 28, 2025. https://www.rpharms.com/Portals/0/RPS%20document%20library/Open%20access/Support/toolkit/pharmaceuticalissuesdosageforms-%282%29.pdf

    • Evrysdi® (risdiplam) film-coated tablets Instructions for Use. Genentech, Inc.

      Evrysdi® (risdiplam) film-coated tablets Instructions for Use. Genentech, Inc.