Avery's Bucket List: Parents' Website For Infant With Spinal Muscular Atrophy …

TUCSON, Ariz., April 25, 2012 /PRNewswire-USNewswire/ — Repligen Corp., in Waltham, Mass., announced today that its experimental drug RG3039, designed to trea.

There is increasing focus on RNA metabolism in motor neuron diseases such as amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, and spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, Jen said.

By Sandra Fish Dinakar Singh, chief executive officer of TPG-Axon Capital Management, discusses his daughter Arya's genetic disease and how it led him to es.

Spinal Muscular Atrophy Daily Mail Spinal Muscular Atrophy : Avery's Bucket List Blog Raises Awareness Of The Huffington Post A newborn baby's battle with.

TUCSON, Ariz., April 25, 2012 /PRNewswire-USNewswire/ — Repligen Corp., in Waltham, Mass., announced today that its experimental drug RG3039, designed to treat spinal muscular atrophy (SMA), was safe and well-tolerated in a phase 1 clinical trial.