Jacobus Pharmaceutical has opened its original, 30-person, invitation-only trial of 3,4-diaminopyridine for Lambert-Eaton myasthenic syndrome (LEMS) to additional participants who meet study criteria
Jacobus Pharmaceutical of Princeton, N.J., is continuing its placebo-controlled trial of 3,4-diaminopyridine (3,4-DAP) for people with Lambert-Eaton myasthenic syndrome (LEMS) who have been receiving the drug through Jacbobus' "expanded access" (also called "compassionate use") program. The study, which began in January 2012, was originally by invitation only.
Many MDA grants for research in myasthenia gravis aim to prevent the immune system from attacking needed muscle proteins while allowing it to fight infection
It can start with nothing more than a drooping eyelid or a slight slurring of speech, either of which can come and go and improve with rest. But it can progress, often gradually over weeks or months, to affect all the voluntary muscles, including those controlling breathing. Weakness may be minimal early in the morning after a good night's sleep and worsen throughout the day, especially after...
Drug testing for Duchenne MD, Leigh syndrome and Pompe disease moves forward; and two MDA-supported mouse studies suggest leads for myasthenia gravis and periodic paralysis
Drug development and identifying new leads for possible drug development are in the news for five neuromuscular diseases in MDA’s program.
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.