Exploring New Avenues for Treating Muscular Dystrophies With Targeted Therapies: Matthew Alexander, PhD
New Study Testing Omaveloxolone in Pregnant Patients, Rozanolixizumab Improves Ocular Symptoms, Efgartigimod to be Studied in Juvenile MG
Pilavapadin Improves Diabetic Peripheral Neuropathic Pain, Application of 2025 McDonald Criteria, FDA Expands Eculizumab Indication to Pediatrics
Clinically Relevant Challenges and Roadblocks of Gene Therapies for Muscular Dystrophies: John Brandsema, MD
FDA Approves Medtronic's Adaptive DBS, Hold Lifted on ELEVATE-44-102 Trial, Ecopipam Meets Primary and Secondary End Points in Tourette
Significance of Recently Approved Mirdametinib for NF1-Associated Plexiform Neurofibromas: P. Leia Nghiemphu, MD