Ultragenyx Pharmaceutical’s Fayuvi is now the first FDA-approved therapy for the underlying cause of Sanfilippo syndrome type A, an ultra-rare inherited neurological disorder. It’s the second approved gene therapy for Ultragenyx, which specializes in rare disease medicines.
Ultragenyx Pharmaceutical’s Genglycos received accelerated FDA approval for glycogen storage disease type Ia, an inherited enzyme deficiency. It’s also the first approved gene therapy for Ultragenyx, which specializes in rare disease treatments.
What if health plans could identify member decline before an avoidable hospitalization occurs? Real-time clinical visibility into long-stay SNF members uncovers risk earlier and drives better outcomes.
Sanofi won European Commission approval for two therapies that treat rare enzyme deficiencies. Xenpoyzme is the first treatment for acid sphingomyelinase deficiency (ASMD) while Nexviadyme is a successor to a blockbuster Sanofi drug for Pompe disease.
Sarepta Therapeutics gains rights to University of Florida-resident startup's programs for central nervous system diseases.