Marisa Wexler, MS, senior science writer —

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is central to NORD’s mission and history — community…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

The first published case of a person developing ocular myasthenia gravis (MG) secondary to COVID-19 was described in a new report. The patient, a 65-year-old woman with a medical history of kidney cancer, pituitary tumor, and a blockage in the pulmonary artery in the lungs, ultimately was successfully…

Treatment with Argenx‘s investigational therapy efgartigimod leads to significant and early reductions in the severity of generalized myasthenia gravis (gMG), regardless of the presence of antibodies against acetylcholine receptor (AChR), according to results from a Phase 3 clinical trial. Trial findings were presented earlier this month at…

Altered levels of immune proteins associated with the complement system may be implicated in generalized myasthenia gravis (MG), a new study suggests. The study, “Changes in serum complements and their regulators in generalised myasthenia gravis,” was published in the European Journal of Neurology. The complement system…

A patient with three different types of myasthenia gravis (MG)-associated antibodies was described in a recent case report. The report, “Anti-MuSK positive myasthenia gravis with anti-Lrp4 and anti-titin antibodies,” was published in Internal Medicine. MG is caused by the immune system attacking the…

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…

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…

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)…

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…