SMA Foundation
Non-Profit & Charitable Organizations · Wyoming, United States · 11-50 Employees
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Overview
Headquarters
970 W Broadway Ste E, Jackson, Wyoming, ...Website
www.smafoundation.orgRevenue
$6.5 MillionIndustry
ZI Rank: 1
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About SMA Foundation
The SMA Foundation is dedicated to accelerating the development of treatments for Spinal Muscular Atrophy (SMA), a leading genetic cause of death in young children. They focus on research and the discovery of drugs, with three FDA-approved treatments currently available and more clinical trials in progress. The foundation aims to support patients and their families affected by SMA. Their intended clients include individuals diagnosed with SMA and their caregivers.Explore more
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SMA Foundation News & Media
Positive Phase 2 Topaz Trial Extension Data Demonstrate Sizable and Sustained Motor Function Improvement at 24 Months with Apitegromab for Non-Ambulatory Patients with Types 2 and 3 Spinal Muscular Atrophy (SMA)
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a Phase 3, clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced new data from the Phase 2 TOPAZ trial extension period evaluating patient outcomes after 24-months of treatment, which support sustained and continued improvement with apitegromab for non-ambulatory patients with Types 2 and 3 SMA receiving an SMN therapy. DetailedBiophytis Presents Preclinical Efficacy Data for Sarconeos (BIO101) in Spinal Muscular Atrophy (SMA), a Rare Neuromuscular Disease
This oral presentation given by Dr. Cynthia Bézier and entitled: "COMBINATION OF BIO101 WITH ANTISENSE OLIGONUCLEOTIDE THERAPY DEMONSTRATES SYNERGISTIC BENEFICIAL EFFECTS IN SEVERE SMA-LIKE MICE" demonstrates, in a mouse model of SMA, the synergic effect of Sarconeos (BIO101) with antisense therapy.New Data Presented at Cure SMA Reveal Residual Unmet Needs in Young SMA Patients Treated With Gene Therapy and Suggest Further Potential of Using SPINRAZA® (nusinersen)
CAMBRIDGE, Mass., June 15, 2022 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) will present new data from clinical studies aimed at assessing remaining unmet needs for people living with spinal muscular atrophy (SMA) and evaluating the potential impact of SPINRAZA® (nusinersen) in different patient populations at the SMA Research & Clinical Care Meeting hosted by Cure SMA this week in Anaheim, Calif. Biogen is a presenting sponsor of Cure SMA’s 2022 Annual SMA Conference, the world’s largestFDA Approves Label Extension for Evrysdi® for Infants with Spinal Muscular Atrophy Under 2 Months Old
- Evrysdi is the first and only at-home administered treatment for patients living with SMA - - Evrysdi has proven efficacy in babies, children and adults with more than 5,000 patients treated to date - SOUTH PLAINFIELD, N.J., May 31, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:...
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The SMA Foundation is dedicated to accelerating the development of treatments for Spinal Muscular Atrophy (SMA), a leading genetic cause of death in young children. They focus on research and the discovery of drugs, with three FDA-approved treatments currently available and more clinical trials in progress. The foundation aims to support patients a... Read More
