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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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Central and Peripheral Treatment of SMA meeting summary

November 20, 2017

On 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…

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Are you a Type II/III SMA Patient or a Caregiver?

April 24, 2017

Help us create App games to measure SMA patients’ motor abilities and strength. Click here for more information.

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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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Spinal Muscular Atrophy Program Advances with Additional Product Candidate Entering Clinical Development

January 7, 2016

-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…

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Breaking News from the Muscular Dystrophy Association: Congress Passes the Ensuring Access to Clinical Trials Act (S. 139/H.R. 209)

September 28, 2015

This 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…

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SMA Foundation Appoints Story Landis to its Board of Directors

March 13, 2015

The 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…

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Every Life Foundation for Rare Diseases issues end of year action alert

December 30, 2014

As 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…

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SMA Foundation comments to FDA on the importance of combination therapy for rare diseases like SMA

December 19, 2014

Last 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…

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Orally delivered compounds, which selectively modify RNA splicing, prevent deficits in mouse models of Spinal Muscular Atrophy

August 7, 2014

Results 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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Congratulations to Dr. Brian McCabe and Dr. Livio Pellizzoni for Their Recent Publications in Cell

October 11, 2012

Congratulations to Dr. Brian McCabe and Dr. Livio Pellizzoni of Columbia University’s Motor Neuron Center for their publications in Cell. The first study, led by Dr. McCabe, shows that SMN restoration in proprioceptive neurons and interneurons corrects defects in motor neurons and muscles in a fly SMA model. Surprisingly, SMN restoration in muscles or motor…

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