Microdystrophin Proposed as a Surrogate End Point in DMDMarch 16th 2023
Jeffrey S. Chamberlain, PhD, Professor and McCaw Chair, Muscular Dystrophy, and director, Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, discussed the potential use of microdystrophin as an end point for clinical evaluations of gene therapies for DMD.
Expert Insights in Hemophilia Gene Therapy for Bleeding Disorders Awareness MonthMarch 14th 2023
In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest expert insights in gene therapies for hemophilia, which is among the most common of these disorders.
ALS Gene Therapy to be Discussed at the 2023 Muscular Dystrophy Association Clinical & Scientific ConferenceMarch 13th 2023
Matthew B. Harms, MD, associate professor of neurology at Columbia University, and medical consultant and care center director at the MDA, discussed the session he will be chairing at the conference.