FDA Approves Alexion’s Ultomiris for Treatment of gMG

On April 28, the US Food and Drug Administration (FDA) granted approval to ravulizumab (Ultomiris) for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody. To date, Ultomiris is the third disease modifying drug approved to treat gMG. Ultomiris will be made available in the United . . .

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Clinical Trial Alert: Phase 2 Study of ALXN2050 in Adults Living with gMG

Researchers at Alexion Pharmaceuticals are seeking adults living with generalized myasthenia gravis (gMG) to participate in a phase 2 clinical trial to evaluate the safety, effectiveness, and pharmacological properties of the investigational drug ALXN2050. ALXN2050 is designed to target a component of the immune system (known as complement), which underlies many autoimmune disorders including gMG. People . . .

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2022 Clinical & Scientific Conference March 15 Highlights

Today was the second full day of sessions of 2022 MDA’s Clinical & Scientific Conference. The three major themes of today’s sessions were (1) therapeutic strategies to treat NMDs, (2) clinical trial design and implementation, and (3) optimizing patient care.  Highlights Mitochondrial interventions: small molecule and gene therapy Carlos Moraes, PhD, from the University of . . .

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2022 Clinical & Scientific Conference March 14 Highlights

Today was the first full day of sessions of MDA’s 2022 Clinical & Scientific Conference. For the first time since the start of the COVID-19 pandemic, clinicians, scientists, policymakers, nonprofit, and industry leaders are convening, primarily in-person, for a dynamic and informative five days in Nashville, TN. With more than 975 in-person and 490 virtual . . .

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FDA Approves argenx’s Vyvgart for Treatment of gMG

On Dec. 17, the US Food and Drug Administration (FDA) granted approval to efgartigimod (Vyvgart) for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody. To date, Vyvgart is the second disease modifying drug approved to treat gMG. Vyvgart will be made available in the United States . . .

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Research Study Alert: Study of New Device for use in Adolescents Living with Autoimmune Neuromuscular Conditions

Researchers at Worrell are seeking adolescents (ages 12 – 17) with the following conditions: neuromyelitis optica spectrum disorder (NMOSD), myasthenia gravis (gMG), myelin oligodendrocyte glycoprotein antibody disease (MOG-AD), or autoimmune encephalitis (AE), to participate in a research study to test the safety and efficacy of a new syringe device being developed to administer a drug to . . .

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Clinical Trial Alert: Phase 1b/2a Study of Descartes-08 in Adults with Generalized Myasthenia Gravis

Researchers at Cartesian Therapeutics are seeking adults living with generalized myasthenia gravis (gMG) to participate in a phase 1b/2a clinical trial to evaluate safety, tolerability and manufacturing feasibility of the investigational drug Descartes-08 to treat gMG. Descartes-08 is designed to eliminate the aberrant plasma cells that cause gMG, potentially reducing the clinical symptoms of the disease. . . .

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Clinical Trial Alert: Phase 3 Study of Rozanolixizumab in Adults with Generalized Myasthenia Gravis

Researchers at The Neurological Institute, PA, located in Charlotte, North Carolina, are seeking adults living with generalized myasthenia gravis (gMG) to participate in a phase 3 clinical trial to evaluate efficacy and safety of the investigational drug rozanolixizumab. Rozanolixizumab is designed to alter the immune response and reduce antibody production, potentially improving strength, fatigue, swallowing, and . . .

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Clinical Trial Alert: Phase 3 Study of Zilucoplan in Individuals With Generalized Myasthenia Gravis

Researchers at Ra Pharmaceuticals (now part of UCB BioSciences) are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study to confirm safety, tolerability, and efficacy of the investigational drug Zilucoplan. This therapy may improve muscle function by blocking the autoimmune reaction that destroys the junction between nerves and muscles in . . .

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Clinical Trial Alert: Phase 3 Study of Rozanolixizumab in Individuals With Generalized Myasthenia Gravis

Researchers at UCB BioSciences are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study. The objective of the study is to confirm the clinical efficacy and to assess safety and tolerability of rozanolixizumab. The therapy may help reduce the typical signs and symptoms of gMG, which are caused by autoantibodies . . .

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