At a NeurologyLive Clinical Forum in Philadelphia, Chafic Karam, MD, chief of neuromuscular medicine at Penn Medicine, discussed treatment sequencing in myasthenia gravis.
Laurie Gutmann, MD, chair of the Department of Neurology at Indiana University School of Medicine, discusses one-year pooled ACHIEVE data for z-basivarsen (DYNE-101), an investigational antisense oligonucleotide, in myotonic dystrophy type 1 (DM1).
Postmarketing reports of product-related insertional oncogenesis involving GLI1 have prompted an FDA investigation into malignancy risk with SMA gene therapy, onasemnogene abeparvovec.
Interim phase 1b data showed that cizutamig produced rapid, sustained improvement in generalized myasthenia gravis with no cases of cytokine release syndrome.