Placebo-treated myasthenia gravis (MG) patients experience as many adverse events as those receiving an active treatment during clinical trials, but this leads to few participant dropouts — and should not represent a problem for patient compliance to placebo during trials, a grouped analysis of studies shows. The study, “…

The presence of autoimmune diseases, such as myasthenia gravis (MG), should not be seen as a not be seen as a factor against treatment with immune checkpoint inhibitors in patients with advanced metastatic cancer, a case report says. The case report, “Metastatic Merkel cell carcinoma…

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…

Patients with myasthenia gravis (MG) who also have other autoimmune diseases do not have a significantly higher disease burden compared to those with MG alone, a study says. The findings of the study, “Impact of autoimmune comorbidity on fatigue, sleepiness and mood in myasthenia gravis,” were published…

Treatment with Rituxan (rituximab) led to high rates of clinical remission without posing significant side effects in a group of patients with myasthenia gravis (MG), a study shows. The study, “High efficacy of rituximab for myasthenia gravis: a comprehensive nationwide study in Austria,” was published in the…

A significant proportion of patients with generalized myasthenia gravis (MG) treated with Alexion’s Soliris (eculizumab) were able to either stop or reduce their use of immunosuppressive therapy, according to interim results from a Phase 3 open-label extension study. The study, “Changes in Concomitant Immunosuppressive Therapy Use…

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies(NCATS) at the…

Treatment with subcutaneous immunoglobulin (SCIg) — a new formulation given as an under-the-skin injection — enabled people with myasthenia gravis (MG) to maintain stable disease activity while transitioning from intravenous immunoglobulin (IVIg), according to a study in the U.S. and Canada. The findings of the study, “…

A new extended release formulation of zilucoplan, a potential self-administered therapy for patients with generalized myasthenia gravis (gMG), given once weekly, achieved complement 5 (C5) inhibition similarly to zilucoplan administered daily, pre-clinical data in non-human primates show. Zilucoplan, developed by Ra Pharmaceuticals, is an artificial peptide that binds to…

With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…