For the first time, a boy receives a commercially available gene therapy for cerebral X-linked adrenoleukodystrophy (ALD) in the United States
2023-05-11
On March 16th, 2023, after thirty years of research started in France with the support of ELA, the first boy in the world received the FDA approved gene therapy for the cerebral form of adrenoleukodystrophy. Following a positive newborn screening, 6-year-olds Conner Hess from New York was followed closely toRead More →
