Catalyst Pharmaceuticals launches Firdapse expanded access program for eligible people living with congenital myasthenic syndromes and Lambert-Eaton myasthenic syndrome
Catalyst Pharmaceuticals, a biopharmaceutical company focused on rare debilitating diseases, announced encouraging results on Sept. 29, 2014, from its phase 3 clinical trial of Firdapse (amifampridine phosphate tablets equivalent to 10mg amifampridine) for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS).
How MDA-supported research to counteract a complement protein and rev up regulatory T cells may improve MG treatment
"I’m on CellCept, prednisone, Mestinon and IVIG every three weeks," says 38-year-old Rachel Pegram. "Prednisone, which I have taken for more than 22 years now, has very rough side effects. It has caused weight gain, diabetes and glaucoma. I cannot say I have ever gone into remission without drugs, but I believe I have been in a drug-induced remission. I have spent a lot of time in hospitals for...
Researchers supported in part by MDA have identified antibodies to the LRP4 protein as a third known cause of myasthenia gravis
A research team supported in part by MDA has shown that an abnormal reaction of the immune system against a protein called LRP4 can be added to the known causes of myasthenia gravis (MG), a disorder involving fluctuating weakness and fatigue because of impaired nerve-to-muscle communication. MG is an autoimmune disease, meaning it's caused by abnormal activity of the body's immune system against...
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
Update (April 14, 2014): This phase 2 trial of 3,4-diaminopyridine is now closed to new participants. However, the drug remains available on a "compassionate use" -- also called "expanded access" -- basis to qualified patients with LEMS and related disorders of the neuromuscular junction. See Treatment Use of 3,4-Diaminopyridine; or enter NCT01765140 in the search box at ClinicalTrials.gov.
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.