IN THE NEWS
ALD: Gene Therapy and the Gift of a Future
An experimental gene therapy developed by Mass General’s Florian Eichler, MD, is giving hope to patients and families impacted by ALD — a fatal brain disease affecting boys under the age of 12.
FDA Advisory Committee Unanimously Endorses eli-cel Gene Therapy for Cerebral Adrenoleukodystrophy
If approved, eli-cel will be the first and only gene therapy for the treatment of early active CALD, a rare neurodegenerative disease that primarily affects young children and leads to irreversible loss of neurologic function and death