Chester County News and Community Website
WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) has announced positive outcomes from a Phase 2 study evaluating QTORIN™ 3.9% rapamycin anhydrous gel for the treatment of microcystic lymphatic malformations …
Palvella Therapeutics Reports Promising Results for QTORIN Rapamycin in Treating Rare Genetic Disease Read More
MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has announced that Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder, will present at the Oppenheimer Movers in Rare Disease Summit in New York City …
Ocugen to Present at Oppenheimer Movers in Rare Disease Summit Read More
WASHINGTON, D.C. — The U.S. Food and Drug Administration (FDA) has announced a groundbreaking approval of Kebilidi (eladocagene exuparvovec-tneq), marking the first gene therapy sanctioned for the treatment of aromatic L-amino …
Revolutionary Gene Therapy Approved: Kebilidi Offers New Hope for Rare Genetic Disorder Read More
WASHINGTON D.C. — The U.S. Food and Drug Administration (FDA) has made a landmark decision to approve Aqneursa (levacetylleucine) for the treatment of neurological symptoms linked to Niemann-Pick disease type C …
FDA Greenlights Aqneursa for Niemann-Pick Disease Type C, Marking a Breakthrough in Rare Disease Treatment Read More 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
LANGHORNE, PA — Savara Inc. (Nasdaq: SVRA) announced that its abstract on inhaled Molgramostim for Autoimmune Pulmonary Alveolar Proteinosis (aPAP) has been accepted for a poster presentation at the 2024 European …
Savara Inc. to Present at European Respiratory Society Congress 2024 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
WASHINGTON, D.C. — This week, in a significant step for the medical world and hope for patients, the U.S. Food and Drug Administration expanded the approval of Elevidys, a gene …
FDA Broadens Approval of Elevidys Gene Therapy for Duchenne Muscular Dystrophy Patients Read More
WASHINGTON, D.C. — On Thursday, U.S. Senators Bob Casey (D-PA) and Markwayne Mullin (R-OK) introduced the Creating Hope Reauthorization Act, a bipartisan effort aimed at encouraging pharmaceutical companies to develop …
Senate Leaders Push for Reauthorization of Program to Combat Rare Pediatric Diseases Read More