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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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Cerebrum podcast features SMA researcher Lee Rubin discussing recent advances in SMA therapeutics

April 5, 2019

SMA researcher and Harvard University professor Lee Rubin was featured in a podcast with Bill Glovin from the Dana Foundation to talk about the recent Cerebrum article he co-authored, “Spinal Muscular Atrophy: Huge Steps.” Both the article and podcast discuss recent advances in SMA therapeutics. To listen to the podcast, please visit: https://soundcloud.com/danafoundation/spinal-muscular-atrophy-huge-steps

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Novartis announces FDA filing acceptance and Priority Review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1

December 3, 2018

Basel, December 3, 2018 – Novartis today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA® (onasemnogene abeparvovec-xxxx), an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1. ZOLGENSMA is designed to address the genetic root cause of…

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Scholar Rock Announces Publication of Preclinical Data on the Therapeutic Benefit of Inhibiting Myostatin Activation in Models of Spinal Muscular Atrophy

December 3, 2018

CAMBRIDGE, Mass., Nov. 27, 2018 (GLOBE NEWSWIRE) — Scholar Rock Holding Corporation (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced the publication, “Specific Inhibition of Myostatin Activation is Beneficial in Mouse Models of SMA Therapy” in the peer-reviewed journal Human Molecular Genetics. The publication details…

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SMA researchers Frank Bennett and Adrian Krainer win the prestigious Breakthrough Prize

October 19, 2018

Excerpted from Breakthrough Prize press release:   A Total of $22 Million Awarded for Breakthroughs in Treatment for the Leading Genetic Cause of Infant Death, Super-Resolution Imaging, Discovery of a New Kind of Electronic Material, and Other Major Achievements.  2019 Breakthrough Prize in Life Sciences Awarded to C. Frank Bennett and Adrian R. Krainer.  All…

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The Spinal Muscular Atrophy Foundation Rings Nasdaq Opening Bell

August 18, 2018

The Spinal Muscular Atrophy (SMA) Foundation, a non-profit, organization whose mission is to accelerate the development of treatments for SMA, the leading cause of genetic death in young children, visited the Nasdaq MarketSite in Times Square on August 17, 2018. In honor of the occasion, Dinakar Singh, Co-Founder and Chairman of the Board of Directors,…

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AveXis to Initiate Screening for Remaining Patients in Pivotal Trial of AVXS-101 for SMA Type 1 Following Review of Preliminary Data from First Three Patients

January 30, 2018

Chicago, Ill. (January 30, 2018) – 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, following review of safety data and early signals of efficacy from the first three patients dosed in its pivotal trial of AVXS-101 for spinal muscular…

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AveXis Announces Expanded Clinical Development Program for AVXS-101in Spinal Muscular Atrophy

January 16, 2018

Chicago, Ill. (January 16, 2018)- AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today provided an overview of the expanded clinical development program for the company’s initial gene therapy candidate, AVXS-101, for the treatment of spinal muscular atrophy (SMA). In addition to…

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AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101

December 13, 2017

Chicago, Ill. (December 13, 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, the company may initiate its planned Phase 1…

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Ionis and Biogen Win Prestigious Prix Galien Best Biotechnology Product Award for SPINRAZA

October 31, 2017

The SMA Foundation congratulates Ionis and Biogen for being awarded the Prix Galien USA Award for the Best Biotechnology Product in 2017 for SPINRAZA® (nusinersen). The Prix Galien USA Award recognizes extraordinary achievement in scientific innovation that improves the state of human health. The award was presented at a ceremony in New York City on…

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Novartis to resume enrollment in ongoing study of branaplam in type 1 SMA

September 29, 2017

Effective immediately, following the respective Health Authorities and Ethics Committee approvals, Novartis will be resuming enrollment in the ongoing study in type 1 SMA (CLMI070X2201) at their existing sites in Belgium, Germany, Denmark and Italy. Please click here to read the full letter.

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