Observed Differences in PIRA Definition Call for More Standardized Agreement on Term
Newborn Screening, Enzyme Delivery, and the Future of Pompe Disease Treatment
AMX0035 Continues to Stabilize and Improve Wolfram Syndrome at 48 Weeks, Phase 2 HELIOS Trial Shows
FDA Clears IND to Study Gene Therapy CAP-002 in STXBP1 Developmental Epileptic Encephalopathy