Chester County News and Community Website
BERWYN, PA — AscellaHealth has released its Q2 2025 Specialty & Rare Pipeline Digest™, offering stakeholders a detailed look at the rapidly evolving landscape of specialty and rare disease therapies. The …
AscellaHealth’s Latest Digest Tracks Surge in Specialty and Rare Disease Drug Approvals Read More
BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) has announced an updated timeline for submitting its Biologics License Application (BLA) for nomlabofusp, a potential first-in-class treatment for Friedreich’s Ataxia …
Larimar Therapeutics Aligns with FDA Guidance, Revises Nomlabofusp Submission Timeline Read More
WEST CHESTER, PA — The West Chester community is gearing up for Step Forward to Cure TSC®, a heartfelt awareness and fundraising event benefiting the TSC Alliance®. Taking place on …
Take a Bold Step to Fight TSC – Join West Chester’s Walk for Hope on Saturday! Read More
WASHINGTON, D.C. — The U.S. Food and Drug Administration (FDA) has announced its recent approval of Ctexli (chenodiol), marking the first-ever treatment for cerebrotendinous xanthomatosis (CTX), a rare and debilitating …
FDA Breakthrough: First-Ever Treatment Approved for Rare and Devastating CTX Disorder! Read More
KING OF PRUSSIA, PA — SEED Therapeutics Inc. has announced that its molecular glue therapy, ST-01156, has been granted Rare Pediatric Disease and Orphan Drug designations by the U.S. Food and …
SEED Therapeutics Secures FDA Rare Pediatric and Orphan Drug Designations for ST-01156 Read More
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
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
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