Gene therapy

Latest Articles

Editorial
Spinal muscular atrophyFurther expanding the clinician’s armamentarium
John F. Brandsema, Charlotte J. Sumner

Neurology September 05, 2019
Clinical/Scientific Notes
Combination therapy with nusinersen and AVXS-101 in SMA type 1
Bo Hoon Lee, Erin Collins, Leann Lewis, et al.

Neurology September 05, 2019
Clinical/Scientific Notes
Fatal TTR amyloidosis with neuropathy from domino liver p.Val71Ala transplant
Ross C. Puffer, Robert J. Spinner, Hongyan Bi, et al.

Neurology Genetics August 14, 2019
Clinical/Scientific Notes
Brain copper storage after genetic long-term correction in a mouse model of Wilson disease
Ricarda Uerlings, Daniel Moreno, Oihana Murillo, et al.

Neurology Genetics May 18, 2018