PLAINSBORO, N.J. — Novo Nordisk announced today that the US Food and Drug Administration (FDA) approved Alhemo® (concizumab-mtci) injection as a once-daily prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or B (HA/HB) without inhibitors, expanding...

NOVATO, Calif. — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the Phase 3 Aspire study evaluating GTX-102 (apazunersen) as a treatment for Angelman Syndrome is fully enrolled, with approximately 129 participants ages four to 17 with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. “The accelerated enrollment of the Phase...

PALO ALTO, Calif. — Neuvivo, Inc., a late-stage biopharmaceutical company developing treatments for neurodegenerative disease, today announced it has gained alignment with the U.S. Food and Drug Administration (FDA) on a regulatory path forward for NP001 as a unique immunotherapy to treat amyotrophic lateral sclerosis (ALS). This follows a Type...