Standardizing Clinical Practice for End of Life Care in Neuromuscular Diseases: Lauren Treat, MD
March 26th 2023The child neurologist at the University of Colorado School of Medicine talked about her presentation on end of life care for patients with neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 5 minutes]
SYMBYX Neuro Light Therapy Wellness Helmet Positively Impacts Parkinson Disease Symptoms
March 25th 2023Over a 12-week treatment period, patients with Parkinson disease on light therapy demonstrated statistically significant improvements of 58% and 40% in lower and upper limb coordination and movement.
New MDA Program for Gene Therapy Development in Ultra-Rare Diseases: Sharon Hesterlee, PhD
March 24th 2023At the 2023 MDA Conference, the executive vice president and chief research officer of MDA talked about a new program from the MDA to tackle ultra-rare neuromuscular diseases. [WATCH TIME: 5 minutes]
Reassessing Treatment Options, Quetiapine for Parkinson Disease Psychosis: Dilesh Doshi, PharmD
March 24th 2023The vice president of Health Economic and Outcomes Research at Acadia Pharmaceuticals provided perspective on the negative effects seen from quetiapine and how the knowledge of pimavanserin has improved since approval. [WATCH TIME: 5 minutes]
Improving Quality of Life, Digestive Health in Duchenne Muscular Dystrophy: David Brumbaugh, MD
March 23rd 2023The associate professor of pediatrics at the University of Colorado School of Medicine talked at the 2023 MDA Conference about how digestive health impacts patients with Duchene Muscular Dystrophy. [WATCH TIME: 4 minutes]
Myasthenia Agent Zilucoplan Continues to Show Positive Efficacy in Long-Term Extension
March 23rd 2023In participants who received placebo during the parent studies, rapid improvements were observed as early as week 1 after switching to zilucoplan, and continued through week 12 of the extension period.
Efforts to Expand Neuropalliative Care for Neuromuscular Disorders: Ambereen Mehta, MD, MPH
March 23rd 2023The palliative care physician and assistant professor of medicine at Johns Hopkins Bayview Medical Center discussed the types of resources clinicians can pull from to expand neuropalliative care for patients with neuromuscular disorders. [WATCH TIME: 3 minutes]
Consensus Considerations Developed for SRP-9001 Adverse Event Management in DMD
March 23rd 2023The 12-person Dephi panel reviewed data from 3 clinical trials of delandistrogene maxeparvovec (SRP-9001; Sarepta), and developed considerations for the management of vomiting, myocarditis, acute liver injury, and immune-mediated myositis.
Turning Gene Therapy Into Reality: Peter Marks, MD, PhD
March 20th 2023The director of the Center for Biologics Evaluation Research at the FDA provided an overview of the Keynote Address he gave at the 2023 Muscular Dystrophy Association Clinical and Scientific Conference. [WATCH TIME: 3 minutes]