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About SMA: SMA Press

Risdiplam meets primary endpoint in pivotal FIREFISH trial in infants with Type 1 SMA

January 23, 2020

The SMA Foundation’s partners, Roche and PTC Therapeutics, announced positive data from the pivotal Part 2 of FIREFISH, a study evaluating risdiplam in infants aged 1-7 months with Type 1 SMA. The study met its primary endpoint of a statistically significant proportion of infants sitting without support for at least five seconds after one year…

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Scholar Rock announces completion of TOPAZ trial enrollment

January 10, 2020

Scholar Rock announced today that enrollment of its TOPAZ Phase 2 trial of SRK-015 in patients with Type 2 and Type 3 SMA is complete. SRK-015 is a selective inhibitor of myostatin that has been shown to increase muscle mass and strength in animal models. For more information, please see the press release from Scholar…

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AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process

September 29, 2017

Chicago, Ill. (September 29, 2017) – AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has notified the company that based on review of data submitted, including the potency assay, it may initiate its…

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Roche to begin Part 2 of the SUNFISH Trial

September 25, 2017

To read the full press release, please click here: SUNFISH

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Scholar Rock to Develop SRK-015 to Improve Muscle Function in Patients with Spinal Muscular Atrophy

June 28, 2017

First data on inhibition of myostatin activation in a preclinical model of SMA to be highlighted at the Cure SMA Annual Conference Treatment with SRK-015 resulted in significantly increased muscle strength in a genetic model of SMA CAMBRIDGE, Mass., June 28, 2017 – Scholar Rock, a biotechnology company focused on discovering and developing drugs that…

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RG7916 Granted Orphan Drug Designation in the U.S. for the Treatment of Spinal Muscular Atrophy

January 6, 2017

SOUTH PLAINFIELD, N.J., January 6, 2017 – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (OOD) to RG7916 for the treatment of patients with Spinal Muscular Atrophy (SMA). RG7916 is part of PTC’s joint development program in SMA with Roche and the SMA…

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PTC Therapeutics Announces Initiation of FIREFISH Study in Infant (Type I) SMA Patients

January 6, 2017

SOUTH PLAINFIELD, N.J., January 5, 2017 – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that its joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation (SMAF) initiated a clinical study in infants with Type I SMA. The study, named FIREFISH, will investigate the safety, tolerability and efficacy of RG7916 in…

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FDA Approves Biogen’s SPINRAZA™ (nusinersen) for Spinal Muscular Atrophy

December 29, 2016

The SMA Foundation is excited to share the news that the U.S. Food and Drug Administration (FDA) approved Biogen’s SPINRAZA™ (nusinersen) under Priority Review for the treatment of Spinal Muscular Atrophy (SMA) in pediatric and adult patients. SPINRAZA is the first and only treatment approved for SMA. (Please see http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm534611.htm for additional information.) SPINRAZA offers…

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Comprehensive Book on Spinal Muscular Atrophy Published

December 1, 2016

We are pleased to announce that the book “Spinal Muscular Atrophy: Disease Mechanisms and Therapy,” has been published by Elsevier and is now available in retail outlets, as well as at the Elsevier Store in print and electronic formats here. This book is a comprehensive reference of recent advances in basic and clinical SMA research…

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AveXis Announces Single-Arm Design for U.S. Pivotal Study of AVXS-101 in SMA Type 1 Patients

November 2, 2016

CHICAGO, Nov. 01, 2016 (GLOBE NEWSWIRE) — AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that the planned pivotal study of AVXS-101 in spinal muscular atrophy (SMA) Type 1 will reflect a single-arm design, using natural history of the disease as…

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Biogen’s Regulatory Applications for Nusinersen as a Treatment for Spinal Muscular Atrophy Accepted by FDA and EMA

October 28, 2016

Biogen today announced that its New Drug Application (NDA) for nusinersen, an investigational treatment for spinal muscular atrophy (SMA), has been accepted by the U.S. Food and Drug Administration (FDA) for Priority Review, and that the company’s Marketing Authorization Application (MAA) has been validated by the European Medicines Agency (EMA). Nusinersen had previously been granted…

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Biogen Submits NDA to the FDA for Nusinersen

September 27, 2016

Biogen and Ionis have taken a crucial step in the pathway to approval of nusinersen. Biogen has completed the submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA) and will submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the coming weeks. Biogen has also applied…

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