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…

NMD Pharma is launching a Phase 1/2a trial in the Netherlands to evaluate for the first time NMD670, an oral treatment candidate for people with myasthenia gravis (MG). The study (CHDR1948), approved by the Dutch ethics committee and regulatory authorities, will take place at the Centre…

A 60-year-old man being treated for multiple myeloma was found to have myasthenia gravis (MG) caused by antibodies against muscle-specific tyrosine kinase (MuSK), a study reports. This unusual case indicates that cancer treatments may mask the symptoms of MG, complicating diagnosis, researchers said. The case was described…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…