News

The Institute for Clinical and Economic Review (ICER), an independent nonprofit that evaluates the prices and benefits of medications in the U.S., reported on the efficacy and cost-effectiveness of Soliris (eculizumab) and efgartigimod. Its report found reasonable evidence that adding either of these medications to conventional care could…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

Soliris (eculizumab) appears to be safe and effective for patients with hard-to-treat generalized myasthenia gravis (gMG) who have acetylcholine receptor (AChR) antibodies, a study has found. Its benefits were extended to a particular group of patients from the Phase 3 REGAIN clinical trial and its extension study who were treated…

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…