Top researcher appointed inaugural Dr Patricia Kailis Chair in Rare Diseases
Internationally recognised leader in rare neuromuscular disease research Professor Gina Ravenscroft has been appointed the inaugural Dr Patricia…
Base editing approach repairs deadly Dravet syndrome mutation in mice
Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially…
GOP Sen. Johnson investigating FDA rare disease drug rejections
WASHINGTON — Sen. Ron Johnson (R-Wis.) said he is investigating the Food and Drug Administration’s rejections of rare disease…
AI model popEVE: Prioritizing genetic variants effectively
By combining deep evolutionary signals with human population data, the popEVE model provides a novel approach to identifying…
GLP-1 goliath updates demand for top weight loss drug
Novo Nordisk reported Q3 2025 earnings on November 5, showing continued strong demand for its weight-loss drug Wegovy,…
Bionano Highlights 9 OGM Studies at ASHG 2025 on SVs
Bionano Genomics (NASDAQ: BNGO) participated in the American Society of…
A Rogue Gene Explains Mysterious Cases of Diabetes and Epilepsy in Newborns
Credit: Wikimedia Commons When six infants around the world were diagnosed with an odd trio of symptoms (diabetes,…
Oxford Nanopore Announces PromethION Plus Flow Cell and Human Genetics Updates at ASHG 2025
OXFORD, England, October 15, 2025–(BUSINESS WIRE)–At the American Society of Human Genetics (ASHG) 2025 industry session on 16th…
Baylor Genetics Presents New Data on Clinical and
HOUSTON, Oct. 15, 2025 (GLOBE NEWSWIRE) — Baylor Genetics, a clinical diagnostic laboratory at the forefront of genetic…
One-size-fits-(nearly)-all fix for V2R mutations
Genetic/congenital Genetic mutations are the primary cause of most rare diseases. Although each condition affects a small fraction…
Saol Therapeutics hit with CRL for rare genetic disease
The U.S. FDA issued a complete response letter (CRL) for the NDA to privately held Saol Therapeutics Inc.’s…