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. …
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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…
From lighting up buildings to issuing local proclamations, efforts are underway to mark Myasthenia Gravis Awareness Month, observed each June for 20 years now. Coordinated annually by the Myasthenia Gravis Foundation of America (MGFA), the campaign is aimed at heightening public awareness of myasthenia gravis (MG), a neuromuscular…
European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…
People who develop ocular myasthenia gravis (OMG) before age 40 are more likely to go into remission after surgery to remove the thymus (thymectomy), according to a Chinese study. The work also supported thymectomy as a safe and effective approach to treat OMG, with about 40% of patients…
Treatment with rituximab is safe, provides clinical improvements and enables stopping maintenance therapies in patients with late-onset myasthenia gravis (LOMG), a small study suggests. The study, “Rituximab in Late‐Onset Myasthenia Gravis is Safe and Effective,” was published in the journal Muscle & Nerve. Rituximab —…
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