Genetic Disorders

Spinal Muscular Atrophy Rare Diseases Sickle Cell Disease Duchenne Muscular Dystrophy Neurofibromatosis Progeria Down Syndrome Gene Therapy Trisomy 18 Epidermolysis Bullosa

Man Who Mistook Prince William for Singer Prince Sends Apology Letter

Sebastian Smith’s family say they helped the 22‑year‑old write to the prince after a Somerset café visit, a note that has drawn attention to local services for adults with learning disabilities and prompted no public reply from Kensington Palace.

Nikon Disqualifies Small World in Motion Winner Over Generative-AI Processing

Nikon Re-Reviews Prize-Winning Microscope Video Over Alleged AI Alterations

Primate Brain RNA BC200 Found Embedded in Human Poxvirus

FDA Approves Isembyld for Spinal Muscular Atrophy

Base Editing Changes Single DNA Letters in Human Embryos but Causes Unpredictable Damage

DeepMind Releases AlphaGenome Atlas Mapping 9 Billion Human DNA Variants

Ultragenyx Phase 3 Angelman Drug Fails to Show Benefit

Belgian Influencer Michiel Vandeweert Dies at 28

Anvisa Registers Duvyzat for Duchenne Muscular Dystrophy

Belgian Influencer With Progeria Michiel Vandeweert Dies at 28

James Buckley Says He Has High Lipoprotein(a) and Faces Elevated Heart Risk

Vertex Raises 2026 Revenue Range on Cystic Fibrosis Drug Strength

Jesy Nelson Shares Rooftop Photo That Prompts Dating Speculation

CRISPR Base Editing Reduces Toxic Huntingtin and Eases Symptoms in Mice

England to Begin National Newborn Screening for SMA in October as an Evaluation

Mainoo Gifts World Cup Semi Ticket to FTD Campaigner

Teen Fan Dies Days After Pawan Kalyan Fulfilled His Final Wish

Carrie Underwood Shares Childhood Photo After Performing At Alan Jackson’s Farewell

Jay & Pamela’s New Season Centers on Their Debate Over Having Children