SUNFISH is a 2-part, randomized, placebo-controlled trial in 231 patients 2 to 25 years of age with later-onset (Type 2 or 3) spinal muscular atrophy (SMA): the first placebo-controlled trial in adults and children older than 9 years with SMA. Purposely designed to include older adults and children with complications, such as contractures and severe scoliosis, participants reflected the real-world SMA population.1,2
*The least square (LS) mean difference for change from baseline in MFM-32 score (95% CI).
†Based on the missing-data rule for MFM-32, 6 patients (n=5 for Evrysdi; n=1 for placebo) were excluded from the analysis.
MFM-32=Motor Function Measure-32 Items.
The validated, 32-item scale is designed to capture meaningful change in gross and fine motor skills among a broad range of ambulatory and nonambulatory people with SMA.
The 32 items of the MFM-32 are divided into 3 domains19,21:
Domain 1
Standing and transfers
13 total items used to identify changes in people with higher motor functions, such as:
Raise pelvis
Sit up
Stand from or sit in chair‡
Squat or reach floor‡
Run or walk‡
Stand from floor
Balance or hop on one foot‡
Domain 2
Axial and proximal limb motor function
12 total items used to monitor a wide range of torso and limb functions, such as:
Raise head‡
Roll over
Raise hands while seated‡
Raise foot
Maintain position without upper limb support‡
Place and extend arms on table‡
Domain 3
Distal limb motor function
7 total items used to identify subtle changes in people with limited motor functions, such as:
Trace‡
Flex foot
Tear paper
Pick up and hold coins or tennis ball‡
Draw continuous loops
MFM-32 items are scored as follows20: 0=does not initiate movement or starting position cannot be maintained; 1=partially completes the exercise; 2=completes the exercise with compensations, slowness, or obvious clumsiness; 3=completes the exercise with a standard pattern. The MFM-32 total score is calculated as a percentage of the maximum score of 96.
*Standard deviation: 11.4 (range, 14.6-66.7).21
†Standard deviation: 18.8 (range, 14.6-93.8).21
‡Some items in this list have been consolidated for simplicity but are scored separately.
*Patients in the placebo arm received placebo for 12 months followed by Evrysdi for 12 months. Evrysdi period not shown.
†Number of patients with an available total score (result) at respective time points. Intent-to-treat patients.
MFM-32=Motor Function Measure–32 Items.
*The least square (LS) mean difference for change from baseline in RULM score (95% CI).
†Based on the missing-data rule for RULM, 3 patients (n=1 for Evrysdi; n=2 for placebo) were excluded from the analysis.
RULM=Revised Upper Limb Module.
This scale assesses the ability to push, pull, place, tear, open, raise, and lift objects, as well as hand, arm, and reaching movements in adults and children.22
The RULM assessment includes 19 scorable items* such as:
Pick up objects, like coins/tokens
Tear paper
Raise cup to mouth
Open plastic container
Nineteen RULM items are scored§ as follows22: 0=unable; 1=able with modification; 2=able without difficulty.
*The RULM includes 20 items, 19 can be scored. (There is an entry item that serves as functional class identification and does not contribute to the total score.)
†Weight is approximate and has been converted from grams to pounds; ~1/2 lb=200 g, ~1 lb=500 g, and ~2 lb=1 kg.
‡Some items in this list have been consolidated for simplicity but are scored separately.
§Of the 19 items, 18 tasks are scored from 0 to 2, while 1 item is scored from 0 to 1.
||Standard deviation: 6.85 (range, 0.0-17.0).
¶Standard deviation: 6.52 (range, 1.0-28.0).
#Standard deviation: 6.89 (range, 10.0-37.0).
**Standard deviation: 3.73 (range, 22.0-37.0).
*Patients in the placebo arm received placebo for 12 months followed by Evrysdi for 12 months. Evrysdi period not shown.
†Number of patients with an available total score (result) at respective time points. Intent-to-treat patients.
RULM=Revised Upper Limb Module.
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.
Chiriboga CA, Bruno C, Duong T, et al. JEWELFISH: safety and pharmacodynamic data in non-naïve patients with spinal muscular atrophy (SMA) receiving treatment with risdiplam. Presentation at: 2020 Virtual CureSMA Conference; June 8-12, 2020; Virtual.
Chiriboga CA, Bruno C, Duong T, et al. JEWELFISH: safety and pharmacodynamic data in non-naïve patients with spinal muscular atrophy (SMA) receiving treatment with risdiplam. Presentation at: 2020 Virtual CureSMA Conference; June 8-12, 2020; Virtual.
Chiriboga CA, Bruno C, Duong T, et al. JEWELFISH: safety and pharmacodynamic data in non-naïve patients with spinal muscular atrophy (SMA) receiving treatment with risdiplam. Presentation at: 2021 Virtual World Muscle Society (WMS) Conference; September 21-24, 2021; Virtual.
Chiriboga CA, Bruno C, Duong T, et al. JEWELFISH: safety and pharmacodynamic data in non-naïve patients with spinal muscular atrophy (SMA) receiving treatment with risdiplam. Presentation at: 2021 Virtual World Muscle Society (WMS) Conference; September 21-24, 2021; Virtual.
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.
Oskoui M, Day JW, Deconinck N, 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). Presentation at: 2021 Virtual MDA Conference; March 15-18, 2021; Virtual.
Oskoui M, Day JW, Deconinck N, 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). Presentation at: 2021 Virtual MDA Conference; March 15-18, 2021; 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.
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.
Clinicaltrials.gov. A study of risdiplam in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (RAINBOWFISH). [NCT03779334]. https://clinicaltrials.gov/ct2/show/NCT03779334. Last update posted June 7, 2021. Accessed June 7, 2021.
Clinicaltrials.gov. A study of risdiplam in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (RAINBOWFISH). [NCT03779334]. https://clinicaltrials.gov/ct2/show/NCT03779334. Last update posted June 7, 2021. Accessed June 7, 2021.
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.
The link you have selected will take you away from this site to one that is not owned or controlled by Genentech, Inc. Genentech, Inc. makes no representation as to the accuracy of the information contained on sites we do not own or control. Genentech does not recommend and does not endorse the content on any third-party websites. Your use of third-party websites is at your own risk and subject to the terms and conditions of use for such sites.