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Foundation News
About Us: Foundation News
The Spinal Muscular Atrophy (SMA) Foundation and PTC Therapeutics have entered a new collaboration focused on regenerative medicine to further advance scientific research in SMA and other neuromuscular disorders. Recent FDA approvals of several disease modifying therapies for SMA has made regenerative medicine approaches the next frontier in drug discovery and development. The SMA Foundation-PTC…
Read MoreThe 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,…
Read MoreOn November 10th, the SMA Foundation held a meeting with over 100 SMA researchers, clinicians, and patient advocacy group representatives to discuss the potential impact of SMN upregulation outside of the central nervous system (CNS). The meeting focused on evidence from preclinical models and patients that indicate SMN has a role outside of the CNS…
Read MoreHelp us create App games to measure SMA patients’ motor abilities and strength. Click here for more information.
Read MoreThe 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…
Read MoreWe 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…
Read More-Phase 1 Study Initiated in Healthy Volunteers with New SMN2 Splicing Modifier – RG7916 – SOUTH PLAINFIELD, N.J., Jan. 7, 2016 /PRNewswire/ — PTC Therapeutics, Inc. (Nasdaq: PTCT) today announced that RG7916, an additional SMN2 splicing modifier from the company’s joint development program with Roche and the SMA Foundation in spinal muscular atrophy (SMA), has…
Read MoreThis afternoon the U.S House of Representatives passed the Ensuring Access to Clinical Trials Act of 2015 (EACT) (S. 139/H.R. 209). Since the U.S. Senate passed the bill in July, the Act is now on its way to President Obama’s desk to be signed into law. EACT makes permanent a law that allows individuals to…
Read MoreThe SMA Foundation has appointed Story Landis to its Board of Directors, effective February 24, 2015. “We are deeply honored to have Dr. Landis join the Foundation’s board. Her commitment to research, therapeutics development and patients is extraordinary,” said Loren Eng, the Foundation’s President. “We look forward to having Dr. Landis’ input and guidance in…
Read MoreAs 2014 comes to a close, we are already preparing for what lies ahead for the rare disease community. Congress has pledged to make biomedical innovation a top-priority in 2015, and it is vital that we use this opportunity to usher in progress for rare disease patients. I’m writing you today with two simple…
Read MoreLast month, the FDA established a public docket for comments on FDA activities performed under the Food and Drug Administration Safety and Innovation Act (FDASIA), Patient Participation in Medical Product Discussions. The SMA Foundation took this opportunity to encourage the FDA to convene a stakeholder meeting on combination therapies, with the goal of producing a…
Read MoreResults published in Science show small molecule SMN2 splicing modifiers to be effective in SMA mouse models. Today the journal Science published results of a preclinical study demonstrating that treatment with orally available RNA splicing modifiers of the SMN2 gene starting early after birth prevents deficits in a mouse model of Spinal Muscular Atrophy (SMA).…
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