Late-Stage Neuromuscular Disorders: MAiD

Akram Khan, MD, FCCP, Associate Professor of Pulmonary, Allergy, and Critical Care Medicine at Oregon Health & Science University, talks about medical assistance in dying (MAiD) regarding late-stage neuromuscular disorders.   Recently, the American College of...

Myasthenia Gravis and Targeted Therapies

Sindhu Ramchandren, MD, Global Clinical Leader at Janssen Pharmaceuticals, explains the pathophysiology of myasthenia gravis and the need for more targeted therapies.   Myasthenia gravis is an autoimmune, neuromuscular disorder characterized by weakness of the...

Targeting SORT1-Positive Cancers

Christian Marsolais, Ph.D., Chief Medical Officer of Theratechnologies, discusses three studies presented at the American Association for Cancer Research (AACR) conference targeting SORT1-positive cancers.   Sudocetaxel zendusortide (TH1902) is a proprietary...

FDA Approves Pegunigalsidase Alfa to Treat Fabry Disease

The U.S. Food and Drug Administration (FDA) has approved pegunigalsidase alfa (Elfabrio), a plant-based enzyme replacement therapy with an initial half-life of 78.9 ± 10.3 hours, for the treatment of adult patients with Fabry disease. Fabry disease is a rare X-linked...