Families of Spinal Muscular Atrophy

This new research funding will support a project at Indiana University School of Medicine to better understand the role of SMN protein in motor axons.

This new research funding will support a project at the University of Southern California to better understand the role of astrocytes in SMA disease pathology.

This new research funding will support a project at Johns Hopkins University to learn more about motor axon growth in mouse models of SMA.

The SMA community now has 3 clinical trials testing new SMA therapies, and an additional 10 programs in earlier stages of the drug development pipeline.

Repligen Corporation has initiated a Phase 1b clinical study of RG3039, a novel small molecule drug candidate for the potential treatment of spinal muscular atrophy (SMA).

Families of Spinal Muscular Atrophy (FSMA) is dedicated to creating a treatment and cure for Spinal Muscular Atrophy (SMA) by funding and advancing a comprehensive research program.

Families of SMA is pleased to announce the award of up to $750,000 for an important new grant to Dr. Brian Kaspar at Nationwide Children’s Hospital.

The 12th annual Concert for a Cure event to be held on May 12th, 2012 is aiming to reach the incredible milestone of raising $1,000,000 for Spinal Muscular Atrophy (SMA) Research.

Repligen Corporation (NASDAQ:RGEN) today announced positive results from a Phase 1 study to evaluate the pharmacokinetic (PK) and safety profile of RG3039, a novel small molecule drug candidate for the potential treatment of SMA.

With the 9th Annual Jacob’s Run, Walk & Roll Event to be held on April 1st, The Jacob Isaac Rappoport Foundation is aiming to reach the milestone of raising $1,000,000 for Spinal Muscular Atrophy (SMA) Research.