Disease registry seeks input from people with congenital muscular dystrophies, congenital myasthenic syndromes or other congenital muscle disorders, including those with no clear diagnosis
An international patient registry (database) is gathering information about children and adults with any form of congenital muscular dystrophy (CMD), a congenital myasthenic syndrome or a congenital myopathy (congenital muscle disease), with the goals of promoting research and improving care in these disorders.
In a phase 2a trial in myasthenia gravis, Cytokinetics’ drug tirasemtiv showed dose-related beneficial effects and no serious adverse events
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...
MDA-supported researchers have used genetic engineering to target renegade cells of the immune system in mice with a disease resembling myasthenia gravis
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...
Inflammation exacerbates muscle destruction in Duchenne muscular dystrophy, and several investigators — including some on the International Space Station — are trying to block it
Inflammation — the immune system's first line of defense in tissue that's been damaged by injury or infection — is a good example of a process that's a good thing up to a point and under certain circumstances and a bad thing in excess or under the wrong circumstances.
Encouraging findings about eculizumab and GM-CSF were presented
The 2012 annual meeting of the American Academy of Neurology, held in New Orleans April 21-28, included findings related to myasthenia gravis (MG).
Top researchers are discussing notable developments in neuroscience, including research in many neuromuscular diseases, at the annual American Academy of Neurology meeting April 21-28
Top scientists and clinicians from around the world are discussing the latest research in neuroscience, and the care of individuals with nerve and muscle diseases, at the 2012 annual meeting of the American Academy of Neurology in New Orleans, April 21-28.
The American Academy of Neurology has released guidelines for the use of intravenous immunoglobulin, which modulates the immune system in MG, LEMS and myositis
The American Academy of Neurology (AAN) has released new guidelines on the use of a treatment called intravenous immunoglobulins (IVIG) in various neuromuscular disorders.