Chester County News and Community Website
PHILADELPHIA, PA — Medicus Pharma Ltd. (NASDAQ: MDCX) has asked the U.S. Food and Drug Administration to grant Rare Pediatric Disease Designation for SkinJect, its experimental treatment for patients with …
Medicus Seeks FDA Rare Disease Status for Skin Cancer Therapy Read More
WASHINGTON, D.C. — The U.S. Food and Drug Administration has approved Bizengri for adults with advanced NRG1 fusion-positive cholangiocarcinoma, marking the first approved treatment for the rare bile duct cancer …
FDA Approves Bizengri for Rare Bile Duct Cancer Read More
WASHINGTON, D.C. — Children with a life-threatening immune disorder now have a new treatment option following federal approval of the first gene therapy designed to correct the underlying cause of …
FDA Approves First Gene Therapy for Rare Childhood Immune Disorder Read More WASHINGTON, D.C. — Children with a rare genetic disorder that affects the brain and body will have access to a newly approved treatment designed to slow disease progression, federal regulators …
FDA Approves First Treatment Targeting Hunter Syndrome Brain Effects Read More
WAYNE, PA — Palvella Therapeutics Inc. (Nasdaq: PVLA) announced the launch of “BEYOND mLM,” a disease awareness campaign and website designed to provide educational resources for patients, caregivers, and healthcare …
Palvella Therapeutics Launches Awareness Campaign for Rare Lymphatic Disease Read More
WASHINGTON, D.C. — The Food and Drug Administration has released draft guidance outlining a new pathway to approve highly individualized treatments for ultra-rare diseases and has granted expedited approval to …
FDA Unveils Ultra-Rare Therapy Path, Fast-Tracks Lung Drug Read More WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) said a newly published systematic review strengthens the scientific case for using topical statins to treat porokeratosis, a rare and progressive genetic …
Topical Statins Show Promise for Rare Skin Disease in New Review Read More 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