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 24, 2017
Biogen will present Phase 3 end of study SPINRAZA® (nusinersen) data from CHERISH, which demonstrated a highly statistically significant and clinically meaningful improvement in motor function in children with later-onset (most likely to develop Type 2 or Type 3) spinal muscular atrophy...
April 21, 2017
On Thursday, May 4, at 1:00pm EST (10:00am PST/11:00am MST/12:00pm CST), Cure SMA will hold a webinar updating the community on the status of Spinraza access. Among the topics covered will be:
- The current status of dosing in states across the US
- Administration sites,...