Brooklyn, who has the genetic disease spinal muscular atrophy, has overcome great odds. / Kevin Pieper/The Baxter Bulletin Kelly Carter, a physical therapist at Child & Youth Pediatric Day Clinic, puts new leg braces on 2-year-old Brooklyn Marie Gould
Objective To gain further insight into disease pathophysiologic process and potential adaptations through investigating whether cortical dysfunction or plasticity is a feature of spinal muscle atrophy (SMA). Design Prospective, double-center
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.
Repligen licensed RG3039 in 2009 from Families of Spinal Muscular Atrophy (FSMA), a patient advocacy organization that funded and directed the preclinical development of RG3039 with an investment of more than $13
(NASDAQ:RGEN) said Wednesday that in a phase 1 study, its experimental treatment for patients with spinal muscular atrophy was well tolerated. Spinal muscular atrophy is a neuromuscular disease that affects about 20000 people in Europe and the United
