Myasthenia Gravis (MG)

Research Briefs: DMD, Leigh Syndrome, MG, Periodic Paralysis, Pompe Disease

Drug development and identifying new leads for possible drug development are in the news for five neuromuscular diseases in MDA’s program.

$13.6 Million in New MDA Grants Promote Understanding, Treatment of Neuromuscular Diseases

The Muscular Dystrophy Association has awarded 44 new grants totaling $13.6 million to advance the understanding and treatment of neuromuscular diseases. The new grants, most of which took effect Feb. 1, encompass a range of diseases covered by MDA’s research program, and they support innovative approaches to basic research and new drug development.

In addition to addressing 16 specific neuromuscular diseases under MDA’s umbrella, the grants also fund research into muscular dystrophy in general, and research into muscle physiology related to neuromuscular disease.

MG — Socrates Tzartos, Ph.D.

Socrates Tzartos, professor of biochemistry at the Hellenic Pasteur Institute in Athens, Greece, was awarded an MDA research grant totaling $345,033 over a period of three years to develop diagnostic tools for diagnosis of the low-density lipoprotein receptor-related protein 4 (LRP4) form of myasthenia gravis (MG).

MG — Muthusamy Thiruppathi, Ph.D.

Muthusamy Thiruppathi, a postdoctoral research associate in microbiology and immunology in the College of Medicine at the University of Illinois at Chicago, was awarded an MDA development grant totaling $180,000 over a period of three years to pursue ways to restore normal immune system function in myasthenia gravis (MG).

MG — David Richman, M.D.

David Richman, professor of neurology at the University of California, Davis, was awarded an MDA research grant totaling $412,500 over a period of three years to study the anti-MuSK myasthenia (AMM) form of myasthenia gravis (MG).

Tirasemtiv Shows Promise in MG

The experimental drug tirasemtiv has shown promise in a phase 2a clinical trial in 32 people with myasthenia gravis (MG). Improvements in general and respiratory muscle function occurred in response to the medication.

Six hours after receiving a single, oral dose of tirasemtiv, study participants showed improvements on a standardized scale of MG severity (the Quantitative MG score) and in forced vital capacity, a measurement of respiratory function.

'Guided Missile' Strategy for MG Shows Promise in Mice

Researchers funded in part by MDA say a gene-based therapy designed to treat myasthenia gravis (MG) has shown promise in mice with an MG-like disease.

The research team was led by Dan Drachman, a longtime MDA research grantee at Johns Hopkins University in Baltimore, who also co-directs the MDA neuromuscular disease clinic at that institution. Drachman, a professor of neurology, has a special interest in MG, which is an autoimmune neuromuscular disease.

MDA Commits $10.7 Million to Neuromuscular Disease Research

The Muscular Dystrophy Association has awarded 33 new grants totaling $10,684,481 to fund research projects focused on uncovering the causes of, and developing therapies for, neuromuscular disease.

MDA's Board of Directors reviewed and approved the new grants based on recommendations from the Association's Scientific and Medical Advisory Committees, and the grants took effect Aug. 1.

MG — JianRong Sheng, Ph.D.

JianRong Sheng, assistant professor in the department of neurology and rehabilitation at the University of Illinois at Chicago Medical Center, was awarded an MDA research grant totaling $317,058 over three years. The funds will help support Sheng's study of potential therapeutic treatments for myasthenia gravis (MG).

MG — Lin Mei, M.D., Ph.D.

MDA awarded a research grant totaling $390,000 over three years to Lin Mei, professor and director of the Institute of Molecular Medicine and Genetics at the Medical College of Georgia, part of Georgia Regents University. The funds will help support Mei’s research on the role of a protein called LRP4 in myasthenia gravis (MG).