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 —…
The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…
Injections of botulinum toxin type A — commonly known as Botox — for cosmetic or medical purposes were linked to transient side effects similar to symptoms of myasthenia gravis (MG) in two people detailed in a case report. The study, “Botulinum toxin injections associated with suspected myasthenia gravis: An…
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