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FDA approval of new gene therapy to treat Duchenne muscular dystrophy grants hope to 4-year-old SoCal boy

A first-of-its-kind gene therapy was approved to treat Duchenne muscular dystrophy, a devastating condition affecting 1 out of 3,500 to 5,000 boys. ....

Los Angeles , United States , Thomas Kargar , Leigh Maria Ramos Platt , Zac Kargar , Nicole Orellana , Children Hospital Los Angeles Neuromuscular Program , Duchenne Muscular Dystrophy , Gene Therapy , Medical Treatment , Hild Patient ,

World's first recipient of Car-T cancer therapy is now 17

Emily Whitehead welcomes news Car-T is available to Irish children with leukaemia ....

Irish Times , Harma Medtech , Emily Whitehead , Killian Hendy , Ublin Ie , Cart Cell Therapy , Hild Patient ,