The US Food and Drug Administration approved the first gene therapy for the treatment of paediatric Duchenne muscular dystrophy on 22 June, saying that the drug, Elevidys, addresses an urgent unmet medical need. About one in every 3,300 boys in the US are affected by DMD which is caused by a defective gene that results in the absence of dystrophin, a protein that helps keep the body’s muscle cells intact. As the disease progresses, life-threatening heart and respiratory problems may occur.
