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Genetic Disorders

Huntington's Disease Mitochondrial Diseases Sickle Cell Disease Spinal Muscular Atrophy Cystic Fibrosis Neurodegenerative Diseases Epidermolysis Bullosa Batten Disease Progeria Sanfilippo Syndrome

Will Forte Decries 'Coyote vs. Acme' Shelving as Ketchup Sets 2026 Release

Forte calls the tax write-off decision a repeat of past cancellations that eclipsed a film he believes is strong.

FDA Rejects Disc Medicine’s Bitopertin for EPP After Questioning Surrogate Endpoint

Eddie Vedder’s EB Documentary ‘Matter of Time’ Debuts on Netflix

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

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

Jesy Nelson Posts New Update on Twins’ SMA Care as Calls Grow for UK Newborn Screening

Jesy Nelson’s SMA Screening Push Wins Government Support

Jesy Nelson Reveals Twins’ SMA Type 1 Diagnoses as Doctors Warn They May Never Walk

Two-Year-Old Laia With Ultra-Rare KARS Syndrome Dies as Brussels Funeral Is Set for Saturday

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

India Unveils BIRSA 101, First Home-Grown CRISPR Therapy for Sickle Cell Disease

Engineered IgA Antibody Enters Kidney Cysts, Dampens PKD Growth Signals in Mice

Swansea Mother Raises Funds to Seek Experimental Options After Son, 4, Is Diagnosed With Sanfilippo

Graeme Souness Fights Back Tears on GMB, Urges Government Action on 'Butterfly Skin' Disease

Iker ‘El Niño Millonario’ Announces Death of His Mother, Miriam Guadalupe Arizpe García

Kidney Organoids Tie APOL1 Risk Variants to Mitochondrial Failure in Podocytes

Gene Therapy Shows 75% Slower Huntington’s Decline in Early Trial

uniQure Gene Therapy Shows 75% Slowing of Huntington’s in Early Trial

Acton Girl, Nearly 4, Begins Lifelong CLN2 Treatment After Diagnosis

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