Chester County News and Community Website
WAYNE, PA — Palvella Therapeutics Inc. (Nasdaq: PVLA) has completed its application for U.S. approval of QTORIN 3.9% rapamycin anhydrous gel to treat microcystic lymphatic malformations, moving the Wayne-based biotechnology …
Palvella Files FDA Bid for QTORIN Rapamycin in Rare Disease Read More
WASHINGTON, D.C. — The Food and Drug Administration last week granted accelerated approval to Ultragenyx Pharmaceutical Inc.’s Genglycos, the first treatment for glycogen storage disease type Ia, after a clinical …
FDA Clears First Gene Therapy for Rare Glycogen Disorder Read More WAYNE, PA — Palvella Therapeutics (Nasdaq: PVLA) has submitted the first portion of its application seeking U.S. approval of QTORIN rapamycin, a treatment for microcystic lymphatic malformations, moving the company …
Palvella Begins FDA Filing for Rare Disease Drug Candidate Read More
LANSDALE, PA — Nine in 10 people affected by ichthyosis and related skin disorders report emotional or psychological effects from their condition, underscoring the broader burden of diseases that currently …
Survey Finds Rare Skin Disorders Carry Heavy Mental Toll Read More
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