Tag: Muscular Dystrophy

Efgartigimod (argenx) is an effective and well-tolerated therapy for patients with generalized myasthenia gravis (gMG), new research suggests. Results from the phase 3, randomized, placebo-controlled ADAPT trial showed that reduction in disease burden and improvement in strength and quality of life in patients with gMG were consistent across four MG-specific scales for those receiving the

“Symptoms, then, are in reality nothing but a cry from suffering organs” – Jean-Martin Charcot Nearly 130 years after his death, Jean-Martin Charcot’s name still resonates with modern neurologists. The famed 19th century French physician made myriad enduring contributions to the field. His eponymous foot condition might be his best known. He also delved into

Healthy muscle cells rely on the protein dysferlin to properly repair the sarcolemmal membrane, a thin specialized membrane that serves a vital role in ensuring that muscle fibers are strong enough and have the necessary resources to contract. Mutations in the DYSF gene that produces this essential protein causes limb girdle muscular dystrophy type 2B