Foundation News

We note with sadness the passing of Dr. Hung Li, a pioneer in Spinal Muscular Atrophy research, in March of this year. Dr. Li was passionate about SMA basic and therapeutic research, committed to high-quality science and doing the right thing for SMA patients and families. His contributions to the field include the introduction of…

NEW YORK, NY – March 27, 2009 – The Spinal Muscular Atrophy (SMA) Foundation, a nonprofit organization dedicated to accelerating the development of a treatment for SMA, is pleased to announce the completion of enrollment for the pilot study of Biomarkers for Spinal Muscular Atrophy (BforSMA). The first subject was enrolled in the BforSMA study…

SOUTH PLAINFIELD, NJ AND NEW YORK, NY – May 12, 2009 – PTC Therapeutics, Inc. (PTC) and the Spinal Muscular Atrophy (SMA) Foundation today announced the expansion of their research collaboration in which the SMA Foundation will provide up to $8.5 million in funding to PTC. This is the second continuation of the collaboration to fund…

The Biomarkers for Spinal Muscular Atrophy (BforSMA) clinical trial is a current pilot study looking to identify potential biomarkers (measures) that can be used to evaluate SMA disease severity and future treatments. Recruitment is ongoing and as of January 29, 2009, 71 subjects have been recruited from 18 clinical sites in the United States and…

Michael J. Astrue, Commissioner of Social Security, today announced the national rollout of the agency’s Compassionate Allowances initiative, a way to expedite the processing of disability claims for applicants whose medical conditions are so severe that their conditions obviously meet Social Security’s standards. For a full list of 50 conditions that have been selected for…

NEW YORK, NY – October 24, 2008 – The Spinal Muscular Atrophy (SMA) Foundation, a nonprofit organization dedicated to accelerating the development of a treatment for SMA, is pleased to announce the launch of a pilot study of Biomarkers for Spinal Muscular Atrophy (BforSMA). The goal of this study is to identify a potential biomarker…

SMA Patient Registry New Website is Here! https://smaregistry.iu.edu/ Indianapolis, IN —October 15, 2008 – After many months of anticipation we are pleased to announce the unveiling of the new International Spinal Muscular Atrophy Patient Registry Website! The Website now allows individuals to participate in the Registry completely through on-line interaction including registering, completing questionnaires, learning about and…

ANN ARBOR, Mich. — Sept. 15, 2008 – Assay Designs and the Spinal Muscular Atrophy Foundation (SMAF) are very pleased to announce a collaborative agreement for development of reagents and assays for SMN (Survival Motor Neuron) protein to expedite drug discovery and development efforts for spinal muscular atrophy (SMA), the leading genetic cause of mortality in…

ST. LOUIS — APRIL 25, 2008 – Today Build-A-Bear Workshop® introduced a new member to its Bearemy’s Kennel Pals® line – the collection of plush dogs that benefit domestic pet programs across the United States. The latest addition, the Black Labrador, was inspired by the story of a beary special Guest, Shea Megale, and her friendship…

The Patient Advisory Group of the International Coordinating Committee (ICC) for SMA Clinical Trials has published a family-friendly set of guidelines for care in spinal muscular atrophy (SMA) – click here to download the guide – to complement the physician guidelines published in August (for more on the physicians’ consensus statement, see The Journal of…