
(AGENPARL) – SILVER SPRING gio 22 giugno 2023 FDA Approves Elevidys, a gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who do not have a pre-existing reason why they cannot receive this therapy.
Fonte/Source: http://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy