News

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…

Ra Pharmaceuticals is enrolling adults with generalized myasthenia gravis (gMG) in RAISE, its global and pivotal Phase 3 trial evaluating the safety and efficacy of zilucoplan, the Muscular Dystrophy Association (MDA) has announced. The company started preparing to launch the RAISE (NCT04115293) study in early…

The U.S. Food and Drug Administration (FDA) is now reviewing an application from Argenx that seeks approval of efgartigimod (ARGX-113) to treat people with generalized myasthenia gravis (gMG). An agency decision is expected on or before Dec. 17. “This is an important milestone for argenx in our…

People with myasthenia gravis (MG) are at greater risk of being hospitalized or dying from COVID-19 than the general public, and should be prioritized for vaccination, researchers report. COVID-19 vaccines are likely to be safe for MG patients, they added, given that “multiple studies suggest that the influenza vaccine…

An international Phase 3 clinical trial assessing the efficacy, safety, and tolerability of rozanolixizumab as a treatment for generalized myasthenia gravis (MG) is currently recruiting participants at 114 study locations. Conducted by UCB BioSciences, the…

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…

A specific haplotype — or genetic signature inherited from one parent — was identified as a risk factor for the development of generalized late-onset myasthenia gravis (LOMG) in the Italian population. A second haplotype was newly identified as having a potential…

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…

Blood levels of certain immune cells, called follicular helper T-cells (Tfh), are abnormally high in patients with myasthenia gravis (MG)  who have autoantibodies against the acetylcholine receptor, a study shows. Study results showed that cells released inflammatory molecules and were linked with more severe disease, supporting their role as a…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…