Jacobus is inviting 30 selected individuals with Lambert-Eaton myasthenic syndrome to participate in a placebo-controlled trial of 3,4-DAP
posted on January 25, 2012 - 6:00am
Jacobus Pharmaceutical of Princeton, N.J., has opened a randomized, placebo-controlled study of the drug 3,4-diaminopyridine (3,4-DAP) in 30 adults with Lambert-Eaton myasthenic syndrome (LEMS) who have been receiving the drug through its expanded access program. Enrollment is by invitation only.
A drug approved in Europe to treat Lambert-Eaton myasthenic syndrome is now being tested in the United States; clinical trial participants sought
posted on November 18, 2011 - 6:00am
BioMarin Pharmaceutical of Novato, Calif., is conducting a multicenter study of 3,4-diaminopyridine phosphate (3,4-DAP), also known as amifampridine phosphate, in adults with Lambert-Eaton myasthenic syndrome (LEMS).
There are eight U.S. trial sites, with additional sites planned for France, Germany, Italy, Poland and Spain.
An MDA-supported open-label trial found the asthma drug benefited 17 of 18 people with either of two forms of congenital myasthenic syndrome
posted on November 9, 2011 - 2:56pm
Researchers have found that the drug albuterol appears to be beneficial in two forms of congenital myasthenic syndrome (CMS) — CMS related to mutations in the collagen Q (colQ) gene and CMS related to mutations in the DOK7 gene.
Research items about Friedreich's ataxia, myasthenia gravis, mitochondrial myopathies, type 1 myotonic dystrophy, gene therapy and gene modification
posted on July 7, 2011 - 10:28am
Edison drugs target FA, mitochondrial diseases
Quest takes a look at gene therapy, 'antisense' and other cutting-edge scientific approaches and how they're being applied to diseases in MDA's program
posted on July 1, 2011 - 4:15pm
Antisense oligonucleotides block flawed genetic instructions
Antisense oligonucleotides — also called antisense, oligos, or simply AONs — are pieces of genetic code that keep other genetic code from being processed. Designed to pair up with a particular sequence of DNA or RNA, AONs can change, block or destroy targeted genetic instructions in a variety of ways.
Reports from the American Academy of Neurology annual meeting about Duchenne, limb-girdle and myotonic muscular dystrophies, dermatomyositis, myasthenia gravis and spinal muscular atrophy
posted on April 20, 2011 - 4:49pm
Below are brief reports and links to more information about neuromuscular disease research presented at the 63rd annual meeting of the American Academy of Neurology (AAN), held in Honolulu April 9-16, 2011.
An MDA-sponsored meeting explored progress in five key therapeutic strategies under development for neuromuscular diseases
posted on March 18, 2011 - 3:30pm
Moving therapeutic strategies from the laboratory to clinical trials and ultimately to the market as treatments was the theme of the MDA National Scientific Conference held March 13-16, 2011, in Las Vegas.
Some 300 people attended the conference, the first in a planned series of such MDA-sponsored meetings that will emphasize new research and current medical care. The majority of presenters and...
