Families of Spinal Muscular Atrophy

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.

Developing new therapies to treat and ultimately cure SMA is the driving force behind Families of SMA. To achieve this goal, Families of SMA aggressively invests in drug development research.

Nutrition Basics: Fostering Health and Growth for Spinal Muscular Atrophy is Families of SMA’s newest release of the SMA Care Series Booklets, which are provided free of charge to all SMA families, as well as SMA Care Providers worldwide.

Isis Pharmaceuticals, Inc. announced today that it has initiated a Phase 1 study of ISIS-SMNRx in patients with spinal muscular atrophy (SMA). Isis is developing ISIS-SMNRx as a potential treatment for all types of SMA.

Awards to help develop new therapies for SMA. These new awards encompass $745,000 for the basic research portion of the $3 Million in new research funding that FSMA announced earlier this year.

The new funding awards will be allocated into three distinct research areas: 1)Basic Research to understand the disease, 2)Drug Discovery to develop new SMA therapies, and 3)Clinical Research to provide the means to test new drugs effectively.

First ever clinical trial approved for SMA with a novel drug specifically designed to treat the disease.