Patricia Inacio, PhD, science writer —

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Soliris (eculizumab) was found in a real-world setting to safely and effectively treat adults with generalized myasthenia gravis (gMG) who had stopped responding to other treatments, according to a post-marketing surveillance study in Japan. The study “Safety and effectiveness of eculizumab in Japanese patients with…

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…

High levels of autoantibodies against acetylcholine receptors (AChR) in the blood of people with thymoma — a tumor of the thymus — can help to identify patients at risk of developing myasthenia gravis (MG) after tumor surgery, a study suggests. Larger tumors, and partial tumor resection might also increase this risk.

Lower frequency of a specific subset of immune cells in the bloodstream may help identify myasthenia gravis (MG) in cases difficult to diagnose, specifically those testing negative for MG autoantibodies, a study suggests. Published in the journal Muscle & Nerve, the study, “Reduced plasmablast frequency is associated with…

Myasthenia gravis (MG) can develop in people with polycythemia vera (PV), a type of blood cancer, even in the absence of thymomas — tumors of the thymus — a study reporting two cases suggests. The study, “Association of polycythemia vera with positive JAK2V617F mutation and myasthenia gravis: A…

A first person has been dosed in a Phase 2 trial evaluating mezagitamab (TAK-079), Takeda Pharmaceutical’s investigational antibody, as a therapy for generalized myasthenia gravis (MG), according to a press release. The trial (NCT04159805), beginning to enroll at select sites across the U.S., Canada, Italy, Poland,…

A Phase 2 clinical trial of Harbour BioMed‘s investigational therapy batoclimab, also known as HBM9161, for myasthenia gravis (MG), has started dosing patients, the company said in a press release. The trial (NCT04346888) — being conducted at the Huashan Hospital in Shanghai, China — is…

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…