June 15, 2017
The spinal muscular atrophy community is celebrating significant progress toward adding SMA to newborn screening panels in two states.
Missouri to Become the First State to Screen for SMA
In Missouri, Senate Bill 50 has been passed by both the Missouri House and Missouri...
June 14, 2017
The first half of 2017 has been an active six months for Cure SMA and SMA advocates. Cure SMA and members of our community have been working with stakeholders groups across multiple settings to ensure our patients’ voices are heard. Below is a summary of the key issues and audiences...
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...