Mutation-Specific Therapy Brings New Hope for Patient With Duchenne Muscular Dystrophy
Mutation-Specific Therapy Brings New Hope for Patient With Duchenne Muscular Dystrophy https://pediatricsnationwide.org/wp-content/uploads/2026/09/070126SC0192_Edit-web-header-1024x484.jpg 1024 484 Abbie Miller https://pediatricsnationwide.org/wp-content/uploads/2023/05/051023BT016-Abbie-Crop.jpg
Justin Nicholoff is the first patient at Nationwide Children’s to receive del-zota through a managed access program. Since research into exon skipping therapies for Duchenne muscular dystrophy (DMD) took off about 15 years ago, Justin Nicholoff has waited for a therapy designed for his specific genetic mutation. In June, his wait ended as he became…