Gene therapy

Latest Articles

Resident & Fellow Section
Bridging the Gap: Gene Therapy in a Patient With Spinal Muscular Atrophy Type 1
Gianluca Costamagna, Alessandra Govoni, Adina Wise, et al.

Neurology November 01, 2022
Research Article
Newborn Screening for Spinal Muscular Atrophy in New York StateClinical Outcomes From the First 3 Years
Bo Hoon Lee, Stella Deng, Claudia A. Chiriboga, et al.

Neurology July 14, 2022
Review
Progress in Amyotrophic Lateral Sclerosis Gene DiscoveryReflecting on Classic Approaches and Leveraging Emerging Technologies
Samuel N. Smukowski, Heather Maioli, Caitlin S. Latimer, et al.

Neurology Genetics April 27, 2022
Research Article
Safety of AADC Gene Therapy for Moderately Advanced Parkinson DiseaseThree-Year Outcomes From the PD-1101 Trial
Chadwick W. Christine, R. Mark Richardson, Amber D. Van Laar, et al.

Neurology October 14, 2021

Dr. Robert Shapiro and Dr. Amynah Pradhan