News

More adults with generalized myasthenia gravis (gMG) who were treated with efgartigimod in a Phase 3 trial experienced minimal or no symptoms than did those given a placebo, an analysis of new study data reported. Other analyses from the ADAPT trial highlighted the potential benefits of efgartigimod for people who are…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

A 60-year-old man with dysphonia — voice issues when trying to speak — was found to have late-onset myasthenia gravis (MG), according to a recent report. Voice changes often come with aging, and this may lead to a delayed or even missed diagnosis of the neuromuscular condition, the researchers…

Robot-assisted thymectomy surgery — a procedure to remove the thymus gland, here done using a robotic system console — eased the disease symptoms of a Colorado man diagnosed with myasthenia gravis (MG). For Mike Oster, a Fort Collins retiree and avid fisherman, the procedure — performed by a cardiothoracic…

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

Bladder problems like urinary incontinence, urgency, and nocturia — waking up frequently to urinate — are common in people with myasthenia gravis (MG), particularly those with late-onset disease, a study found. Late-onset MG, or LOMG, is defined as disease symptoms that begin after age 50, while symptoms in early-onset…

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

A recent case report describes an elderly woman who was first diagnosed with myasthenia gravis (MG) after experiencing a myasthenic crisis while being sedated for a surgical procedure. “This case reports a form of discovery of [a] myasthenic crisis outside the usual circumstance,” its authors wrote. “The prognosis depends…

The first participant has been dosed in a Phase 3 clinical trial testing the efficacy and safety of batoclimab (HBM9161) in people with generalized myasthenia gravis (gMG) in China. “The first patient first dosing in the phase III study brings us one step closer to deliver the novel therapeutic…

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…