Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy – Pharmacy Today, American Pharmacists Association, pharmacist.com
Medical Xpress |
Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy
Pharmacy Today, American Pharmacists Association, pharmacist.com Lenti-D gene therapy was administered to 17 boys, all in the early stages of disease, and an interim analysis was performed at median followup of 29.4 months. Of the 17 patients, 15 achieved the primary outcome, with only minimal clinical symptoms. Stem cell gene therapy safe for adrenoleukodystrophy |
