Genetic Disorders

Spinal Muscular Atrophy Rare Diseases Sickle Cell Disease Duchenne Muscular Dystrophy Neurofibromatosis Progeria Gene Therapy Down Syndrome Cystic Fibrosis Huntington's Disease

Nikon Re-Reviews Winning Microscopy Video After Questions Over AI Use

The review will determine whether post-processing altered the scientific validity of the prizewinning cilia footage.

Primate Brain RNA BC200 Found Embedded in Human Poxvirus

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

Sickle Cell Disease in Brazil: Screening and Free Treatment Fall Short of Comprehensive Care

World Sickle Cell Day 2026 Focuses on Closing the Survival Gap

Pawan Kalyan Visits Bedridden Teen in Hanumakonda and Provides Rs 1 Lakh for Treatment