Genetic Disorders

Rare Diseases Spinal Muscular Atrophy Sickle Cell Disease Gene Therapy Neurofibromatosis Trisomy 18 Mitochondrial Diseases Prader-Willi Syndrome Epidermolysis Bullosa Angelman Syndrome

Colombian Influencer Alejo Little Dies at 33 as Cause Remains Undisclosed

Fellow creator Carlos Feria announced the death on Instagram after weeks of limited posts.

Unrepaired DNA‑Protein Crosslinks Drive Inflammation‑Linked Premature Aging, Study Finds

ICE Denies Release for Texas Detainee to Attend Son’s Funeral as Family Pursues Legal Reopening

Jesy Nelson and Zion Foster Reportedly Split, Continue Co-Parenting

Josefine Scholl, 17, Reveals Rare McCune-Albright Syndrome on TikTok

UK Mother Who Lost Husband and Son to Rare Alagille Syndrome Leads Awareness Push

Jesy Nelson Sets Feb. 13 Debut for Prime Video Series as She Details Twins’ SMA Type 1 Diagnoses

After Husband and Son Die in Sleep, UK Mother Leads Alagille Syndrome Awareness Push

Becky Quick Reveals Daughter’s SYNGAP1 Diagnosis as CNBC Launches Rare-Disease Initiative

Zimbabwe Captain Sikandar Raza Mourns Death of 13-Year-Old Brother in Harare

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