Gene Therapy May Benefit Patients With Cerebral ALD
An infusion of autologous hematopoietic stem cells appears to halt disease progression and preserve neurologic function.
Neurology Reviews. 2018 January;26(1):33
December 11, 2017|Neurology Reviews
—Erik Greb
Suggested Reading
Eichler F, Duncan C, Musolino PL, et al. Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy. N Engl J Med. 2017;377(17):1630-1638.
Engelen M. Optimizing treatment for cerebral adrenoleukodystrophy in the era of gene therapy. N Engl J Med. 2017;377(17):1682-1684.
