News

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…

Almost 90% of patients diagnosed with myasthenia gravis develop other diseases, or comorbidities, while about 70% have treatment-related complications, a review study from India reports. These additional disorders may contribute to a poorer outcome for these people, making early recognition and management of comorbidities essential for improving patient care and overall…

A combination therapy of high-dose cholinesterase inhibitors, such as Mestinon, with calcineurin inhibitors, such as Prograf, increases the chances of muscle cramps in patients with Myasthenia Gravis, thereby reducing their quality of life, a Japanese study has found. The study, “Treatment of Myasthenia Gravis…

Researchers have identified five candidate therapies for treating myasthenia gravis (MG) by using a new computational model that also predicts key biological processes involved in the disease. The study, “Building the drug-GO function network to screen significant candidate drugs…

Ra Pharmaceuticals is preparing to launch a Phase 3 clinical trial to assess the therapeutic potential of zilucoplan for the treatment of patients with generalized myasthenia gravis (gMG). This comes after the company’s completion of End-of-Phase 2 interactions with the U.S. Food and Drug Administration (FDA). The 12-week study — a single, pivotal,…

Robotic surgical removal of the thymus seems to be safe and effective and may reduce or eliminate the need for steroid therapy in myasthenia gravis (MG) patients older than 60 at onset. The study, “Results of robotic thymectomy performed in myasthenia gravis patients older than 60 years at…