W. Arnold

Single‐Dose Gene‐Replacement Therapy for Spinal Muscular Atrophy

Brian K. Kaspar, Lindsay Alfano, J. Mendell, 2017, The New England journal of medicine.

P.372 AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: end-of-Study event free survival and achievement of developmental milestones

J. Kissel, L. Rodino‐Klapac, B. Kaspar, 2017, Neuromuscular Disorders.

AAV1.NT-3 gene therapy attenuates spontaneous autoimmune peripheral polyneuropathy

M. Yalvaç, W. Arnold, W. Arnold, 2015, Gene Therapy.

Mutant small heat shock protein B3 causes motor neuropathy

W. Arnold, T. Prior, J. Kissel, 2010, Neurology.

AVXS-101 Phase 1 Gene Replacement Therapy Clinical Trial in SMA Type 1: Continued Event Free Survival and Achievement of Developmental Milestones (S29.001)

J. Mendell, T. Prior, J. Kissel, 2018, Neurology.

480. Gene Therapy for Spinal Muscular Atrophy Type 1 Shows Potential to Improve Survival and Motor Functional Outcomes

J. Mendell, T. Prior, J. Kissel, 2016 .