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Personalized CRISPR Therapy Sends Baby KJ Home from CHOP

FDA fast-tracked the six-month custom CRISPR therapy that corrected KJ’s CPS1 mutation, enabling normal protein metabolism with reduced drug support.

Simple Finger Test Raises Awareness of Ehlers-Danlos Syndromes and Respiratory Risks

Gene Therapy Restores Immune Function in Children with Rare Disorder

Megan King’s Skull Reattached to Spine After 37 Surgeries Following Rare Injury

Winnipeg Jets Prospect Chaz Lucius Retires at 21 Following Ehlers-Danlos Diagnosis

Fran Jones Recovers After Collapsing During Bogota Tennis Match

FDA Approves VYKAT XR, First Therapy for Hyperphagia in Prader-Willi Syndrome

First In-Womb Treatment for Spinal Muscular Atrophy Shows Promising Results