News

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…

A review of studies related to COVID-19 and neuromuscular disorders such as myasthenia gravis (MG) found that the novel coronavirus can exacerbate symptoms in patients, including those on immune-related therapies.  The review, “Neuromuscular Complications in…

High levels of kappa free light chain — a protein that makes up a portion of immunoglobulins (antibodies) — in the bloodstream may indicate the presence of myasthenia gravis (MG) in patients who are harder to diagnose, a study has found. That includes those testing negative…

Argenx is planning to submit an application to the U.S. Food and Drug Administration (FDA) by the end of this year for its lead candidate, efgartigimod (ARGX-113), for the treatment of generalized myasthenia gravis (gMG), the company announced. Assuming the biologics license application (BLA)…

Soliris (eculizumab) may be a promising treatment option for patients with refractory myasthenia gravis (MG) associated with a tumor in the thymus gland, a case report suggests. The case report study, “Eculizumab as a promising treatment in thymoma-associated myasthenia gravis,” was published in…

Treatment with Soliris (eculizumab) led to rapid and sustained improvements in muscle strength and the ability to perform daily activities in people with refractory generalized myasthenia gravis (gMG) who have antibodies against acetylcholine receptors (AChRs), the Phase 3 REGAIN clinical trial and its…

Stress and depression are associated with higher relapse rates in people with myasthenia gravis (MG), according to a recent study. Attention to evidence of either disorder is important for proper patient care, its researchers said. The study, “Prospective study of stress, depression and personality in myasthenia…