Beyond being a diagnostic tool, antibodies against acetylcholine receptors (AChR) in the blood reflect disease severity and could be a useful biomarker for myasthenia gravis (MG) progression, a study suggested. The findings also indicated that a blood protein called C5a may help identify patients…
Treatment with IMVT-1401 significantly eased symptoms and lowered antibody levels in adults with moderate-to-severe generalized myasthenia gravis (MG), top-line results of a small Phase 2a trial show. Use of this investigative antibody at two doses was also seen to be safe and well tolerated. Results are expected to…
Infusions of intravenous immunoglobulin (IVIG) given before high-dose prednisone safely prevents most of its associated exacerbations in people with generalized myasthenia gravis (MG), a study reports. The study, “…
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…
Johnson & Johnson will acquire Momenta Pharmaceuticals, which has been developing nipocalimab (M281) for the treatment of myasthenia gravis (MG). The process, which involves an all-cash deal totaling about $6.5 billion, is expected to be completed before the end of this year. The acquisition will enable Janssen, a…
Measuring levels of a protein known as suPAR in the blood could be useful for assessing the severity of myasthenia gravis (MG), a pilot study reports. The study, “Serum level of soluble urokinase plasminogen activator receptor (suPAR) as a disease severity marker of myasthenia gravis: a…
The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…
Firdapse (amifampridine phosphate), an investigational treatment for MuSK-antibody positive myasthenia gravis (MuSK-MG), failed to meet its primary or secondary goals, according to top-line results from a Phase 3 clinical trial. The randomized trial (NCT03304054…
Rituximab, an approved treatment for certain cancers and autoimmune conditions, appears to also be effective and well tolerated in people with juvenile myasthenia gravis (JMG) who fail to respond to available therapies, a small study suggests. But before…
When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience. Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…
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