Current Status of Gene Therapies in Rare Neuromuscular Disorders Alan Beggs, PhD and Julie A Parsons, MD

Current Status of Gene Therapies in Rare Neuromuscular Disorders Alan Beggs, PhD and Julie A Parsons, MD Drs. Beggs and Parsons discuss the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at the MDA 2025 conference in Dallas, Texas, in March 2025 and is intended for healthcare professionals only. This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established. In contents of this podcast, shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The ASPIRO clinical trial is on clinical hold since September 2021. https://checkrare.com/current-status-of-gene-therapy-in-rare-neuromuscular-disorders/