For Patients and Caregivers

JEWELFISH

Ongoing commitment to SMA research in patients with previously treated SMA4

An open-label trial studying safety and tolerability4

174 adults, children, and infants (1 to 60 years of age) with Type 1, 2, or 3 SMA enrolled

  • This trial is investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of Evrysdi in both ambulatory and nonambulatory patients
    • Ambulatory and nonambulatory patients: 9% (n=15) walkers, 57% (n=100) sitters, and 34% (n=59) nonsitters
  • Participants have received previous treatment with approved or investigational therapies,* including an SMN2-splicing modifier (≥90 days prior to screening) or gene-replacement therapy (≥12 months, prior to screening)

*Investigational at the start of JEWELFISH. All but 3 patients enrolled in JEWELFISH received previous treatment; these 3 patients were previously enrolled in the MOONFISH trial, received placebo, and were never switched to RG7800.4,46

Including a broad range of age, type, and disease severity4

The study population was heterogeneous with a high degree of motor impairment at baseline

PREVIOUS TREATMENT
  RG7800* (MOONFISH)
(n=13)†		 Nusinersen
(n=76)‡		 Olesoxime*
(n=71)		 Onasemnogene abeparvovec
(n=14)§		 All patients
(N=174)
Patient characteristics4,46
Age at enrollment, years Median (range) >18 years, % (n) 30.0 (16-58)
85% (11)		 12.0 (1-60)
28% (21)		 16.0 (11-36)
44% (31)		 2.0 (1-5)
0%		 14.0 (1-60)
36% (63)
Gender, % (n) Male 69% (9) 53% (40) 49% (35) 79% (11) 55% (95)
SMA type, % (n) 1
2
3		 0%
39% (5)
62% (8)		 12% (9)
57% (43)
32% (24)		 3% (2)
70% (50)
27% (19)		 29% (4)
71% (10)
0%		 9% (15)
62% (108)
29% (51)
SMN2 copy number, % (n) 1
2
3
4		 0%
8% (1)
46% (6)
46% (6)		 0%
13% (10)
72% (55)
15% (11)		 0%
1% (1)
92% (65)
7% (5)		 21% (3)
7% (1)
71% (10)
0%		 2% (3)
8% (13)
78% (136)
13% (22)
Disease severity4
Scoliosis, % (n) Yes
>40° curvature		 69% (9)
23% (3)		 84% (61/73)||
37% (27/73)||		 93% (66)
51% (36)		 27% (3/11)||
0%		 83% (139/168)||
39% (66/168)||
Hip subluxation or dislocation, % (n) Yes 15% (2) 34% (25/73)|| 28% (20) 36% (4/11)|| 30% (51/168)||
Motor function assessment scores at baseline4

Motor function at baseline, % (n)		 Nonsitters
Sitters
Walkers		 54% (7)
23% (3)
23% (3)		 28% (21)
57% (43)
16% (12)		 41% (29)
59% (42)
0%		 14% (2)
86% (12)
0%		 34% (59)
57% (100)
9% (15)
Baseline HFMSE total score <10, % (n) Yes 62% (8) 48% (35/73)|| 83% (59) 27% (3/11)|| 63% (105/168)||

*Investigational therapies; not approved by FDA for any use.46
†In the MOONFISH trial, 3 patients were treated with placebo and were not switched to RG7800.4
‡Three patients had also received olesoxime previously.4
§One patient received treatment with onasemnogene abeparvovec first, followed by nusinersen. Ten patients were enrolled in STRONG, three patients in STR1VE, and one patient in STR1VE EU prior to enrollment in JEWELFISH.4
||Only reported for patients aged 2 to 60 years.4
For patients younger than 2 years, baseline motor milestones were evaluated by the Hammersmith Infant Neurological Examination–Module 2.4

FDA=Food and Drug Administration; HFMSE=Hammersmith Functional Motor Scale Expanded; SMN2=survival motor neuron 2.

Understanding Evrysdi®️ and SMA support brochure icon

Overview of results from our clinical trial program

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Administering and storing Evrysdi

Non-invasive options to fit your patients’ needs1,21

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How Evrysdi works

 Increasing SMN protein production throughout the body1

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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 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.

    • 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.

    • 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

    • 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.

    • 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.