Treatment with immunosuppressive agents and Mestinon (pyridostigmine) protects against transition from ocular myesthenia gravis (MG) to a generalized form of the disease among patients with anti-acetylcholine receptor (AchR) antibodies, according to new research. Women, current or former smokers, and patients with thymus hyperplasia (enlargement) or…
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…
DAS Therapeutics has acquired GTP-004, an investigational oral treatment for myasthenia gravis, from GT Biopharma, the companies announced. GTP-004 will be named DAS-MG from now on, DAS Therapeutics said on its website. The investigational treatment combines a fixed dose of pyridostigmine — an approved therapy for myasthenia gravis-associated muscle…
Physicians should keep in mind that severe exacerbations in patients with myasthenia gravis (MG) might be triggered by the use of gabapentin (sold under the brand name Neurontin, among others), normally used to control seizures, a case report study says. “Uncontrolled recurrent myasthenia gravis exacerbations secondary…
The diabetes drug metformin, which is largely safe and minimally toxic, can be of therapeutical value for myasthenia gravis, according to a study conducted using a mouse model of the disease. The study, “Metformin attenuates autoimmune disease of the neuromotor system in animal models of myasthenia gravis,” was…
Assessing altered eye movements with a technique called electrooculography (EOG) may become a non-invasive method for early diagnosis of myasthenia gravis (MG), according to new research. The study, “Analysis of Electrooculography Signals for the Detection of Myasthenia Gravis,” was published in the journal Clinical Neurophysiology.
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to zilucoplan, Ra Pharmaceuticals‘ lead candidate for the treatment of myasthenia gravis (MG). This status helps to support the potential therapy’s development by granting Ra Pharma certain incentives. Zilucoplan, formerly known as RA101495, is an…
Scientists discovered that patients with myasthenia gravis have a unique metabolic profile that can be used to diagnose the disorder, but also to predict the course of the disease and possibly lead to personalized treatments. The study, “Beyond the antibodies: serum metabolomic profiling of myasthenia gravis,” recently was…
Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…
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