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Gene Therapy

Sarepta Reports 3-Year EMBARK Data Showing Elevidys Slows Duchenne Progression

The readout lands under heightened FDA scrutiny following two liver‑failure deaths in non‑ambulatory patients.

Exa‑cel Posts Complete Primary-End Point Responses in Early Pediatric Trials, With One Fatal Busulfan Event

Gene Therapy Slows Huntington’s Progression by 75% in Early Trial, uniQure Says