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SMA Press


About SMA: SMA Press

FDA Approves label Extension for Evrysdi for Infants with SMA Under 2 Months Old

May 31, 2022

The SMA Foundation’s partners, Roche and PTC Therapeutics, announced today that the FDA has approved a label extension for Evrysdi(risdiplam) to include babies under two months old with SMA. The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that pre-symptomatic babies treated with Evrysdi for at least one…

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Scholar Rock shares community update on SAPPHIRE, a clinical trial evaluating apitegromab in non-ambulatory patients with Type 2 and 3 SMA

November 30, 2021

Today Scholar Rock shared a community update on SAPPHIRE, a clinical trial evaluating apitegromab in non-ambulatory patients with Type 2 and 3 SMA: Dear SMA Community, We are excited to announce the design of SAPPHIRE, a Phase 3 clinical trial to further evaluate the safety and efficacy of apitegromab in those living with Spinal Muscular…

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Spinal Muscular Atrophy Foundation Receives CNF Advocacy Award of Merit

November 10, 2006

New York, NY – November 10, 2006 – The Spinal Muscular Atrophy (SMA) Foundation announced today that it has received the 2006 Advocacy Award of Merit from the Child Neurology Foundation (CNF). The award was presented to the SMA Foundation during the Annual Meeting of the Child Neurology Society in Pittsburgh on October 20. Founded…

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Lexicon Genetics Awarded Additional Grant for Study of SMA

October 11, 2006

The Woodlands, Texas – October 11, 2006 – Lexicon Genetics Incorporated (Nasdaq: LEXG) announced today that its research program to identify targets that may be important in the development of drugs to prevent or treat spinal muscular atrophy (SMA) has been extended for an additional year by the United States Army Medical Research & Materiel…

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SMA Foundation Announces Collaboration with PTC Therapeutics

June 7, 2006

New York, NY – June 7, 2006 – The Spinal Muscular Atrophy Foundation today announced that they have initiated a research and development collaboration with PTC Therapeutics, Inc. (PTC). The collaboration is designed to leverage PTC’s proprietary Gene Expression Modulation by Smallmolecules (GEMS) technology to identify and develop new small molecule therapeutics for use in…

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New Push for NIH Funding Urged

May 20, 2006

By Jonathan D. Rockoff Sun Reporter WASHINGTON, D.C.— May 20, 2006 – Senators urged research advocacy groups and universities yesterday to step up efforts to win more federal funding for the National Institutes of Health, the federal agency that funds most of the country’s medical research but is facing a third consecutive year of cuts.…

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The Today Show, December 26, 2005, Featuring the SMA Foundation

December 26, 2005

On Monday, December 26, 2005, NBC’s The Today Show featured one family’s fight against thgenetic motor neuron disease Spinal Muscular Atrophy and the impact SMA has on parensearching for a treatment for their child. The show highlighted the daily struggle that 6-year-old Arya faces battling the effects of SMA and how she and her parents,…

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New Motor Neuron Center Will Tackle Neurodegenerative Diseases

December 23, 2005

The birth of any organization dedicated to reducing human suffering is fueled by hope and propelled by the desire to achieve extraordinary goals; this was certainly true on Nov. 2 when Columbia’s Motor Neuron Center was launched. The gathering of about 40 leading researchers and clinicians from different disciplines at CUMC and Morningside, committed to…

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Drug Provides Glimmer of Hope for Children Suffering Fatal Genetic Disease

September 14, 2005

Stanford University News by Krista Conger Ching Wang, MD, PhD, didn’t sign up for his pediatric neurology residency in 1990 to watch children die. But, as in nearly any medical specialty, there are some fatal diseases for which no effective treatment exists. Frustrated after delivering grim news to one too many sets of parents, Wang…

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SMA Foundation Makes the Case for Research in Rare Disease

September 1, 2005

By Debbie Strickland The errant gene has been isolated, the National Institutes of Health has made the disease a priority and now a young foundation is pitching biotech companies to develop a cure for spinal muscular atrophy, a disabling, often fatal genetic disorder that afflicts some 25,000 Americans. Dinakar Singh and Loren Eng launched the…

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SMA: Race Is On to Conquer Devastating Childhood Disease

August 1, 2005

SMA: Race is on to Conquer Devastating Childhood Disease When a young child is slow to meet developmental milestones such as rolling over, crawling, taking his first steps – or perhaps reaches them and then slows or slides back in development – parents grow concerned and look for explanations. For about 1 out of every…

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Forbes: For Arya

March 28, 2005

-by Robert Langreth A Wall Street Power Couple Race to Find a Cure for Their Daughter Before It Is Too Late Arya Singh was born in March 2000, sat up and crawled on schedule but never really walked right. Pediatricians said at first that she was simply a late bloomer. But at 17 months she…

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