© 2021 MJH Life Sciences and Neurology Live. All rights reserved.
© 2021 MJH Life Sciences™ and Neurology Live. All rights reserved.
Vamshi K. Rao, MD, and Eric P. Hoffman, PhD, discuss how dystrophin relates to Duchenne Muscular Dystrophy, current approved therapies, and treatments in development.
May 13th 2021
Vamshi K. Rao, MD, and Eric P. Hoffman, PhD, review Duchenne muscular dystrophy and the mutations involved.
Discussion of micro-dystrophin, its place in gene therapy, and relevance in the clinical setting in relation to Duchenne muscular dystrophy.
May 20th 2021
Consideration of the measurement and standardization of dystrophin improvement to infer clinical benefit and the FDA distinction between exon skipping dystrophin and micro-dystrophin in Duchenne muscular dystrophy.
Eric P. Hoffman, PhD, outlines the variables in measurement of dystrophin levels in patients with Duchenne muscular dystrophy and how they translate into clinical benefit.
May 27th 2021
Discussion on the importance of early treatment in patients with Duchenne muscular dystrophy.
A discussion with parents on treatment options in Duchenne muscular dystrophy as well as the benefit of using steroids combined with other therapies.
June 3rd 2021
A review of exon skipping agents, their function, mechanism of action, and the different drugs used in clinical practice to treat Duchene muscular dystropy.
Viltolarsen phase II study data is discussed as well as a comparison of viltolarsen to golodirsen for treatment of Duchenne muscular dystrophy.
June 10th 2021
Recent approval of casimersen, its administration, efficacy, safety, and patient selection for treatment of Duchenne muscular dystrophy.
Final thoughts from leading experts on the future of treating Duchenne muscular dystrophy and keeping standard of care in the forefront.