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Genetic Disorders

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

Nick Jonas Marks Maya Kibbel’s 31st Birthday With Emotional Tribute

The tribute underscores a bond he described as family.

Theo Colker, Grandson of Choreographer Deborah Colker, Dies at 14

Sildenafil Shows Early Benefit in Leigh Syndrome as Europe-Wide Trial Is Planned

Maya Kibbel, Jonas Brothers’ Childhood Friend, Dies at 30 as Nick Jonas Pays Tribute

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

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