Data from: Assessment of disease progression in dysferlinopathy – a one year cohort study

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关键词：

Outcome research;LGMD 2B;DYSF;Dysferlinopathy;Muscular dystrophy;Neuromuscular Disease

DOI：

doi:10.5061/dryad.tp08m60

摘要：

r 6 months and 1 year was calculated and compared between disease severity (ambulant (mild, moderate or severe based on a-NSAA score) or non-ambulant (unable

Data from: Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis

负责人：

关键词：

Clinical Neurology;Clinical Trials;Myasthenia;Autoimmune diseases;Neuromuscular Disease

DOI：

doi:10.5061/dryad.4hk2039

摘要：

consistently demonstrated that 75% showed a rapid and long-lasting disease improvement. Conclusions: Efgartigimod was safe and well-tolerated