Gene therapy

Latest Articles

Article
Adult MTM1-related myopathy carriersClassification based on deep phenotyping
Benjamin T. Cocanougher, Lauren Flynn, Pomi Yun, et al.

Neurology September 20, 2019
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