The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

People with myasthenia gravis who have autoantibodies targeting the proteins LRP4 and agrin have more severe disease compared to patients without those  antibodies, a study has found. However, regardless of antibody status, all patients responded to standard MG therapies. …

3P Biopharmaceuticals and Toleranzia are continuing their partnership to develop Toleranzia’s potential therapy candidate, TOL2, for people living with myasthenia gravis (MG). “Currently there are enormous opportunities offered by biotechnology as we can help to develop treatments for rare diseases or those without current therapeutic approaches, such as…

Immunovant is preparing to launch a Phase 3 trial of its investigational candidate IMVT-1401 in patients with myasthenia gravis (MG); it may lead to the treatment’s approval for this indication, pending positive results. The new trial will build on data from two previous studies of other drug candidates that…

People with myasthenia gravis (MG) who fail to respond to standard therapies or are dependent on steroid use can safely and effectively be treated with rituximab, an immune modulating medicine, a real-world study found. Patients taking rituximab were also…

Defects in mitophagy — the process that degrades defective mitochondria, the cell compartments responsible for producing energy — in certain types of immune cells may cause myasthenia gravis (MG), a study contends. The findings also suggest that an antibiotic with immunosuppressant properties, called rapamycin, holds promise as…

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…

Rituximab seems to be more effective in newly-diagnosed patients with generalized myasthenia gravis (gMG), compared to people at more advanced stages of the disease, a study has found. Investigators also noted that among newly-diagnosed patients, rituximab seems to be superior to other conventional immunosuppressive regimens. The study, “…

Nipocalimab (M281) led to a rapid and sustained response at all four dosing regimens, while causing significant reductions in disease severity among people with generalized myasthenia gravis (gMG), top-line results of the Phase 2 Vivacity-MG clinical trial show. The investigational therapy, developed by Momenta Pharmaceuticals, also showed a…

Achieving minimal disease symptoms six months after thymectomy predicts favorable long-term outcomes in people with myasthenia gravis (MG), a study suggests. The research also found that shortness of breath prior to surgery is associated with worse treatment outcomes. The study, “Prognostic Analysis of Thymoma-Associated Myasthenia Gravis (MG) in…