Chester County News and Community Website
WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) reported positive topline results from a mid-stage clinical trial that positions its lead drug candidate as a potential first approved treatment for …
Rare Disease Breakthrough? Palvella Reports Strong Phase 2 Results Read More
NEW CASTLE, DE — BioTek reMEDys is urging expanded, year-round awareness and education surrounding rare autoinflammatory diseases—conditions that arise when the body’s immune system mistakenly attacks healthy tissues, leading to …
BioTek reMEDys Calls for Year-Round Awareness and Education on Rare Autoinflammatory Diseases Read More
MALVERN, PA — Endo, a wholly-owned subsidiary of Mallinckrodt plc, has introduced the first and only FDA-approved generic version of Amgen’s RAVICTI® (glycerol phenylbutyrate) oral liquid in the United States, …
Endo Launches First Generic Version of Amgen’s RAVICTI, Expanding Access for Rare Disease Patients Read More
PHILADELPHIA, PA and GORDONVILLE, PA — A new study from Children’s Hospital of Philadelphia (CHOP) and the Clinic for Special Children has revealed that complement factor I (CFI) deficiency, an …
Study Finds Ultra-Rare Genetic Disorder Far More Prevalent in Old Order Amish Read More
WASHINGTON, D.C. — The Social Security Administration has added 13 new conditions to its Compassionate Allowances list, a program designed to fast-track disability claims for individuals with the most severe …
SSA Expands Compassionate Allowances List to Speed Disability Claims Read More
WASHINGTON, D.C. — The U.S. Food and Drug Administration (FDA) has approved Papzimeos (zopapogene imadenovec-drba), marking the first approved medical therapy for adults with recurrent respiratory papillomatosis (RRP), a rare …
FDA Approves First Immunotherapy for Rare Respiratory Papillomatosis Read More
BERWYN, PA — Specialty pharmacy and healthcare services firm AscellaHealth is spotlighting its role as the HUB partner for Abeona Therapeutics (Nasdaq: ABEO) in the launch and commercialization of ZEVASKYN™, …
AscellaHealth Drives Patient-Centric Launch Support for Groundbreaking Gene Therapy ZEVASKYN Read More 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