Chester County News and Community Website
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
MALVERN, PA — Endo, Inc. this week unveiled a new television commercial and disease awareness campaign, titled “Reminders,” aimed at empowering patients to advocate for their treatment preferences for Dupuytren’s …
Endo Launches “Reminders” Campaign to Empower Patients with Dupuytren’s Contracture Read More
LANGHORNE, PA — Savara Inc. (Nasdaq: SVRA), a biopharmaceutical company focused on rare respiratory diseases, is set to present groundbreaking research at the upcoming American Thoracic Society (ATS) 2024 International …
Savara Inc. to Highlight Advances in Rare Lung Disease Research at ATS 2024 Read More