May 11, 2017
The Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children (SACHDNC) announced that they have accepted spinal muscular atrophy into the review process for the Recommended Uniform Screening Panel (RUSP). The RUSP is a list of conditions that all newborns in the US...
April 25, 2017
SMA Community's Voice Heard “Loud and Clear” at Last Week's Patient Focused Drug Development Meeting with the FDA
On April 18, 2017, the SMA community—families, clinicians, researchers, industry and regulators—gathered for a Patient-Focused Drug Development (PFDD) Meeting with the FDA.
As part of the reauthorization of the Prescription Drug User Fee Act (PDUFA), the FDA is required to...
April 11, 2017
The SMA community is eagerly looking forward to next week’s Patient-Focused Drug Development (PFDD) Meeting with the FDA.
Over 400 members of the community are registered to participate in this event. This includes individuals and families affected by SMA, representatives from...