scAAV9 Gene Therapy Safe, Effective in Preclinical Study in Epileptic SLC13A5 Deficiency
May 13th 2021The safety profile of the SLC13A5 vector was “excellent” according to study authors, with no adverse effects on weight, general activity, or survival in knockout mice and wild type littermates.
Patient Conversations About Clinical Development in ALS: Matthew B. Harms, MD
May 10th 2021The associate professor of neurology at Columbia University and medical consultant and care center director at the Muscular Dystrophy Association spoke to the importance of staying up on the latest developments in ALS and keeping patients in the loop on that information.
The Impact of Big Data on ALS Care: Matthew B. Harms, MD
May 9th 2021The associate professor of neurology at Columbia University and medical consultant and care center director at the Muscular Dystrophy Association discussed the use of larger datasets to improve prognostication and clinical trials in ALS.