Foundation News

SOUTH PLAINFIELD, NJ – December 6, 2007 – PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of small-molecule drugs targeting post-transcriptional control mechanisms, today announced an expanded research collaboration with the Spinal Muscular Atrophy (SMA) Foundation. The collaboration builds on the existing research agreement to identify and characterize compounds that…

Neurology Today: SMA Drug Development Gains Momentum Under NINDS Model for Neuro-Research Partnerships. Reprinted with permission from the American Academy of Neurology from Neurology Today, Nov. 6, 2007.

The SMA Foundation warmly thank Megan and Shea Megale for all their efforts in raising awareness for children living with SMA. For more information about a recent book signing event in New York City, please see this article in USA Today and the Megales’ interview on the Today show.

New York, NY and Waltham, MA – September 18, 2007 – The Spinal Muscular Atrophy Foundation and BG Medicine today announced a collaboration to discover plasma biomarkers of drug efficacy for spinal muscular atrophy (SMA), the leading genetic cause of mortality in infants and toddlers. This project seeks to discover a clinically-useful molecular biomarker, which can…

March 13, 2007 — Cold Spring Harbor, New York – RNA splicing antisense technology studied at Cold Spring Harbor Laboratory (CSHL) effectively corrected an mRNA splicing defect found in spinal muscular atrophy (SMA) patients, and is now ready to be tested in mouse models. “SMA patients who suffer from motor-neuron degeneration may benefit from our…

Spinal muscular atrophy (SMA) is an inherited disease characterized by the selective death of motor neurons, resulting in generalized muscle weakness that is often fatal in infancy or early childhood. SMA is caused by deletions or mutations in the gene encoding survival motor neuron 1 protein (SMN1), whose function is unclear. SMN1 contributes to the…

MCB is pleased to announce that Kevin Eggan, Assistant Professor of Molecular and Cellular Biology is a recipient of this year’s MacArthur “genius” award in recognition of his stellar contribution to stem cell research. Eggan expressed his gratitude and appreciation for the award: “Needless to say, I was shocked, surprised, overwhelmed, the whole spectrum of…

New York, NY – December 1, 2006 – The Spinal Muscular Atrophy Foundation announces the addition of Ivan Lieberburg, Ph.D., M.D. and Edward Kaye, M.D. to its Scientific Advisory Board. They join a distinguished group of business and scientific leaders who help formulate and execute the SMA Foundation’s drug discovery programs. Currently, there is no…

The Woodlands, Texas — May 11, 2006 – Lexicon Genetics Incorporated (Nasdaq: LEXG) announced today that it was awarded a grant from the United States Army Medical Research & Materiel Command (USAMRMC) for the identification of targets that may be important in the development of drugs to prevent or treat spinal muscular atrophy (SMA), a neurodegenerative…

WASHINGTON, DC – May 16, 2010 – At a time when the cost of developing a new drug is skyrocketing and research money is tight, it is important to be creative in designing new therapies for rare diseases and bringing them to market, according to speakers at the eighth Annual Meeting of the American Society for…