For Patients and Caregivers

Extensively studied, with a consistent safety profile across all approved doses1,28,30

Extensively studied, with a consistent safety profile across all approved doses1,28,30

Patients living with SMA
SUNFISH PART 2
Adverse reactions occurring in ≥5% of adults and children receiving Evrysdi and with an incidence of ≥5% compared with placebo (N=180)1,2
  Evrysdi
(n=120)		 Placebo
(n=60)
Adverse reaction
Fever* 22% 17%
Diarrhea 17% 8%
Rash† 17% 2%
Mouth and aphthous ulcers 7% 0%
Arthralgia 5% 0%
Urinary tract infection‡ 5% 0%

As of clinical cut-off date: September 6, 2019.

  • The most common adverse reactions reported in ≥10% of adults and children receiving Evrysdi and at an incidence greater than placebo were fever, diarrhea, and rash1

*Includes pyrexia and hyperpyrexia.

†Includes rash, erythema, rash maculopapular, rash erythematous, rash papular, dermatitis allergic, and folliculitis.

‡Includes urinary tract infection and cystitis

SUNFISH PART 2
Safety observations over 48 months2
  Placebo
0-12 months
(n=60)§		 Evrysdi
0-12 months
(n=120)		 Evrysdi
12-24 months
(n=120)		 Evrysdi
24-36 months
(n=120)		 Evrysdi
36-48 months
(n=120)
Most common adverse events,|| % (n)
Upper respiratory tract infection 30.0%(18) 31.7% (38) 16.2% (19) 8.6% (10) 17.9% (20)
Cold 25.0% (15) 25.8% (31) 22.2% (26) 9.5%(11) 5.4%(6)
Fever 16.7% (10) 20.8% (25) 13.7% (16) 9.5% (11) 8.0% (9)
Headache 16.7% (10) 20.0% (24) 10.3% (12) 7.8% (9) 3.6% (4)
Diarrhea 8.3% (5) 16.7% (20) 8.5% (10) 6.0% (7) 3.6% (4)
Vomiting 23.3% (14) 14.2% (17) 13.7% (14) 6.9% (8) 7.1% (8)
Cough 20.0% (12) 14.2% (17) 10.0% (12) 4.3% (5) 2.7% (3)
Most common serious adverse events, % (n)
Pneumonia 3.3% (2) 8.3% (10) 6.8% (8) 1.7% (2) 2.7% (3)
Influenza 0% (0) 0.8% (1) 0.9% (1) 0.9% (1) 0.9% (1)

Clinical cut-off date for Evrysdi 0-12 months and placebo 0-12 months: September 6, 2019.

Clinical cut-off date for Evrysdi 12-24 months: September 30, 2020.

Clinical cut-off date for Evrysdi 24-36 months: September 6, 2021.

Clinical cut-off date for Evrysdi 36-48 months: September 6, 2022.

§Patients in the placebo arm received placebo for 12 months followed by Evrysdi. The patients who started in the placebo group and switched to Evrysdi are not included in the 12- to 24-month and 36-month, and 36- to 48-month data.

||Adverse reactions have an assumed causality to drug treatment under appropriate use; adverse events include any untoward event associated with the use of a drug, regardless of causality.

  •  No treatment-related adverse events leading to withdrawal or treatment discontinuation over 48 months16

Some patients may have temporarily stopped taking their Evrysdi and then restarted the treatment. One patient withdrew from the trial after the September 6, 2021 clinical cut-off date because of an adverse event (transaminitis) that was initially reported as related to Evrysdi, and then reassessed after discontinuation as unrelated to Evrysdi.

The most common adverse reactions* to Evrysdi in infantile-onset SMA with an incidence of at least 10% were fever, diarrhea, rash, upper respiratory tract infection (including nasopharyngitis, rhinitis), lower respiratory tract infection (including pneumonia, bronchitis), constipation, vomiting, and cough1

FIREFISH PART 1 AND PART 2

Adverse events* occurring in ≥10% of infants receiving Evrysdi (N=62)2
All-patient cohort (N=62)

  Evrysdi
(N-62)
Adverse event
Upper respiratory tract infection (URTI)† 82%
Pyrexia 55%
Lower respiratory tract infections (LRTI)‡ 53%
Rash§ 29%
Constipation 26%
Diarrhea 19%
Vomiting 18%
Cough 13%
Teething 13%

As of clinical cut-off date: November 12, 2020.

*Adverse reactions have an assumed causality to drug treatment; adverse events include any untoward event associated with the use of a drug, regardless of causality.

†URTI includes COVID-19, influenza, nasopharyngitis, pharyngitis, respiratory tract infection, respiratory tract infection viral, rhinitis, upper respiratory tract infection, upper respiratory tract infection bacterial, viral upper respiratory tract infection.

‡LRTI includes bronchiolitis, bronchitis, COVID-19, lower respiratory tract infection, lower respiratory tract infection viral, pneumonia, pneumonia aspiration, pneumonia bacterial, pneumonia staphylococcal, pneumonia viral, respiratory syncytial virus bronchiolitis, tracheitis, tracheobronchitis.

§Rash includes rash, dermatitis, dermatitis acneiform, dermatitis allergic, erythema, folliculitis, rash maculopapular, rash papular.

  • No treatment-related adverse events leading to treatment discontinuation over 24 months27¶

As of clinical cut-off date: November 12, 2020.

Some patients may have temporarily stopped taking their Evrysdi and then restarted the treatment.

  • Initial safety findings from JEWELFISH have been consistent with the safety findings in FIREFISH and SUNFISH30
RAINBOWFISH
  • 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
  • Adverse events observed in presymptomatic SMA were consistent with those observed in infantile-onset and later-onset SMA1
  • No treatment-related adverse events leading to discontinuation*

As of clinical cut-off date: July, 2021.

*Some patients may have temporarily stopped taking their Evrysdi and then restarted the treatment.

Most common adverse events* (reported in ≥3 infants)28
  Evrysdi
(n=18)
Adverse events, % (n)
Teething 33% (6)
Nasal congestion 28% (5)
Pyrexia 28% (5)
Diarrhea 22% (4)
Viral infection 22% (4)
Vomiting 22% (4)
Constipation 17% (3)
Cough 17% (3)
Eczema 17% (3)

As of clinical cut-off date: July 1, 2021

  • Multiple occurrences of the same adverse event in an individual are counted only once
  • Includes adverse events with onset from first dose to the cut-off date

*Adverse reactions have an assumed causality to drug treatment under appropriate use; adverse events include any untoward event associated with the use of a drug, regardless of causality.

PREVIOUS TREATMENT
  RG7800*
(MOONFISH)(n=13†)		 Nusinersen
(n=76)		 Olesoxime*
(n=70)		 Onasemnogene abeparvovec
(n=14)		 All patients
(N=173‡)
Adverse events occurring in ≥8% of patients,§ % (n)
Pyrexia 15% (2) 30% (23) 16% (11) 43% (6) 24% (42)
Upper respiratory tract infection 0 22% (17) 23% (16) 29% (4) 21% (37)
Headache 8% (1) 22% (17) 19% (13) 0 18% (31)
Nasopharyngitis 15% (2) 16% (12) 11% (8) 36% (5) 16% (27)
Diarrhea 0 22% (17) 9% (6) 7% (1) 14% (24)
Nausea 0 18% (14) 10% (7) 7% (1) 13% (22)
Cough 0 16% (12) 9% (6) 21% (3) 12% (21)
Serious adverse events occurring in >2% of patients, % (n)
Pneumonia 0 4% (3) 1% (1) 7% (1) 3% (5)
Respiratory failure 0 4% (3) 1% (1) 0 2% (4)
Respiratory distress 0 3% (2) 0 7% (1) 2% (3)
Lower respiratory tract infection 0 1% (1) 3% (2) 0 2% (3)
Upper respiratory tract infection 0 4% (3) 0 0 2% (3)

As of clinical cut-off date: January 31, 2022.

*Investigational therapies; not approved by FDA for any use.

†In the MOONFISH trial, 3 patients were treated with placebo and were not switched to RG7800.30

‡One patient withdrew from the study at baseline; therefore, 173 patients received Evrysdi.

§Multiple occurrences of the same adverse event in one individual are counted only once.

  • Evrysdi treatment duration in months, median (range):  26.8 (0.9-59.0)
  • As follow-up duration is different between groups, the overall rate of adverse events and serious adverse events cannot be compared
  • The safety findings from JEWELFISH have been consistent with the safety findings in FIREFISH and SUNFISH
No required monitoring or laboratory testing

When initiating Evrysdi, it is important to be aware of any medications your patient is currently taking. This includes prescription medications (such as statins), over-the-counter medications (such as stool softeners), vitamins, dietary supplements, or additional fiber that may impact bowel movements

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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;384:915-923. doi: 10.1056/NEJMoa2009965.

      Baranello G, Darras BT, Day JW, et al. Risdiplam in Type 1 spinal muscular atrophy. N Engl J Med. 2021;384:915-923. 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.