Avery's Bucket List: Parents' Website For Infant With Spinal Muscular Atrophy …
A newborn baby's battle with spinal muscular atrophy inspired her parents to start a bucket list blog. HULIQ reports on Avery's Bucket List blog, a site that aims to spread awareness of Avery's illness, which progressively
Repligen has reported positive results from Phase I study of RG3039 for the potential treatment of spinal muscular atrophy (SMA). The blinded, ascending, single dose study demonstrated that RG3039 was well tolerated at all doses administered and showed
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.
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 establish and fund the Spinal Muscular Atrophy Foundation. You've probably never heard of Spinal
If you were asked which genetic disease is the number one cause of death among babies in the United States, chances are spinal muscular atrophy would