Chester County News and Community Website
WASHINGTON, D.C. — The U.S. Food and Drug Administration (FDA) has approved Miplyffa (arimoclomol) for the treatment of Niemann-Pick disease, type C (NPC), marking the first drug sanctioned to address …
FDA Approves Miplyffa for Niemann-Pick Disease, Type C Read More
KING OF PRUSSIA, PA — The Association for Frontotemporal Degeneration (AFTD) announced a major fundraising success, thanks to a $25,000 challenge match from the Milburn Foundation. The “Empowered Together to #EndFTD” …
AFTD’s Campaign Exceeds Goals, Raises $75,000 for FTD Research and Support Read More
PHILADELPHIA, PA — Passage Bio, Inc. (NASDAQ: PASG) has announced a series of agreements to out-license its three clinical-stage pediatric lysosomal storage disease programs to GEMMA Biotherapeutics, Inc. (GEMMABio). The …
Passage Bio Out-Licenses Rare Disease Programs to GEMMA Biotherapeutics Read More