Genetic Disorders

Rare Diseases Sickle Cell Disease Gene Therapy Neurofibromatosis Trisomy 18 Angelman Syndrome Epidermolysis Bullosa Down Syndrome Duchenne Muscular Dystrophy Huntington's Disease

Grey’s Anatomy Alum Steven W. Bailey Discloses Rare CMS Diagnosis, Plans Wheelchair-Inclusive Roles

He says the rare neuromuscular disorder has caused progressive fatigue, leading to greater reliance on a powered wheelchair.

Hilary Swank Privately Apologizes After LAX Run-In With Make-A-Wish Mom

Capricor’s HOPE-3 Trial Reports Cardiac and Muscle Gains in Duchenne Muscular Dystrophy

Regeneron, Tessera Strike $150 Million Gene-Editing Pact for Alpha‑1 Antitrypsin Deficiency

TV Audience Awards Reported 25 Million Pesos to Help 5-Year-Old Twins With Noonan Syndrome

Altman, Mulherin and Armstrong Back $30 Million Embryo-Editing Startup Preventive

Katie Price Renews Appeal to Dwight Yorke With Video of Harvey in Man United Shirt

Intellia Pauses Phase 3 Trials of CRISPR Therapy After Severe Liver Event

Isabelle Tate, Actress in '9-1-1: Nashville,' Dies at 23

A Year After Sammy Basso’s Death, Parents Reveal Letters as Research He Championed Presses On

Scientists Map Precise Breakpoint Driving Human Robertsonian Chromosome Fusions

Acadia Halts Carbetocin for Prader-Willi After Phase 3 Miss

FDA Grants Accelerated Approval to Forzinity, First Therapy for Barth Syndrome

High Court Rules £100,000 Raised for Kyle Will Go to DIPG Charities

Custom CRISPR Base Editor Corrects ACTA2 Mutation in Mice, Extending Survival Fourfold

Nature Study Reveals How RAP1 Blocks DNA-PK to Safeguard Telomeres

CRISPR Study Ties DENND1A Regulatory Variants to Elevated Testosterone in PCOS

Mariners Capture Inaugural Vedder Cup as Raleigh Belts 50th

Unapei Survey Flags Inadequate Schooling for Disabled Children Ahead of French School Start

Israel Releases Records Disputing Daily Mirror ‘Starving Child’ Cover