Defects in mitophagy — the process that degrades defective mitochondria, the cell compartments responsible for producing energy — in certain types of immune cells may cause myasthenia gravis (MG), a study contends. The findings also suggest that an antibiotic with immunosuppressant properties, called rapamycin, holds promise as…
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The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…
Rituximab seems to be more effective in newly-diagnosed patients with generalized myasthenia gravis (gMG), compared to people at more advanced stages of the disease, a study has found. Investigators also noted that among newly-diagnosed patients, rituximab seems to be superior to other conventional immunosuppressive regimens. The study, “…
Nipocalimab (M281) led to a rapid and sustained response at all four dosing regimens, while causing significant reductions in disease severity among people with generalized myasthenia gravis (gMG), top-line results of the Phase 2 Vivacity-MG clinical trial show. The investigational therapy, developed by Momenta Pharmaceuticals, also showed a…
Achieving minimal disease symptoms six months after thymectomy predicts favorable long-term outcomes in people with myasthenia gravis (MG), a study suggests. The research also found that shortness of breath prior to surgery is associated with worse treatment outcomes. The study, “Prognostic Analysis of Thymoma-Associated Myasthenia Gravis (MG) in…
A new type of immunotherapy successfully eliminated immune B-cells that wrongly target the MuSK protein in people with MuSK-associated myasthenia gravis (MG), a study in mice found. While the treatment effectively killed damaging B-cells, it spared healthy cells, setting the stage for its possible testing in people. …
The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.
As part of Myasthenia Gravis (MG) Awareness Month, Argenx — the biotech company developing efgartigimod to possibly treat this disease — has opened a patient-focused platform called MG United. The company unveiled the online platform on June 1 during its virtual MG Illuminate event, which included segments on doctor-patient…
Argenx’s lead candidate efgartigimod (ARGX-113) safely and effectively eases symptoms in people with generalized myasthenia gravis (gMG) who have acetylcholine receptors (AChR) autoantibodies, top-line data from a Phase 3 clinical trial show. While most of the trial’s goals were focused on AChR-positive patients (the most common group in…
While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…