Genetic Disorders

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

Belgian Influencer With Progeria Michiel Vandeweert Dies at 28

His death prompted public tributes and a planned public funeral on KRC Genk’s stadium field.

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

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

Scientists Report First Use of Base Editing in Human Embryos

Genome-Wide Molecular Timer Controls Developmental Stages in C. elegans

Four-Year-Old Héctor Dies After Battle With Infantile Tay‑Sachs

Daniel Coleman Announces Death of His 14‑Year‑Old Son

Jesy Nelson Marks Twins’ First Birthday With Renewed Push for SMA Newborn Screening

NICE Backs Routine NHS Use of Two SMA Drugs as England Secures Lifelong Access

Inherited POT1 Mutations That Lengthen Telomeres Tied to Eightfold Higher Lymphoma Risk

Zolgensma Given to SMA Infant in Hyderabad After Minister Helps Close Funding Gap

Ancient DNA Study Finds Recent Selection Shaped Hundreds of Human Genes

Human Neuron Study Links Early Synapse Overactivity to Sanfilippo Dementia