Progress in ALS research requires a free flow of information between MDA-supported laboratory investigators and leaders in the drug-development industry
ALS, also known as amyotrophic lateral sclerosis and Lou Gehrig’s disease, is among the most feared diseases facing people in the U.S. and worldwide. In ALS, the motor neurons — the nerve cells of the brain and spinal cord that control muscle action — mysteriously deteriorate, often within just a few years, leaving behind orphaned muscle fibers. Without signals from motor neurons, voluntary...
MDA's 2015 Scientific Conference points the way toward tomorrow's therapies
Last month, MDA brought together more than 400 of the nation’s leading scientists, researchers and professionals specializing in neuromuscular disease research and care at its 2015 Scientific Conference in Washington, D.C., March 11–14. This pre-eminent gathering, hosted every other year, was designed to accelerate discoveries and drug development for families fighting neuromuscular diseases.
Research and clinical trial updates
MDA welcomes two new muscle disease and ALS experts
As part of MDA’s bold plan to accelerate treatments and cures for neuromuscular diseases, two new scientific program officers have joined the MDA family: Amanda Haidet-Phillips, Ph.D., and Laura Hagerty, Ph.D. Under the leadership of MDA Senior Vice President and Scientific Program Director Grace Pavlath, Ph.D., they will...
Santhera Pharmaceuticals is testing anti-cell death compound omigapil in children and teens with merosin-deficient and Ullrich congenital muscular dystrophies
Santhera Pharmaceuticals continues to recruit participants for a phase 1 trial of omigapil in children and adolescents with the type 1A form of congenital muscular dystrophy (CMD)
Israel-based BioBlast Pharma says a California site will be added soon to locations already testing Cabaletta in oculopharyngeal muscular dystrophy in Israel and Canada
BioBlast Pharma, based in Tel Aviv, Israel, has been given clearance from the U.S. Food and Drug Administration (FDA) to test its experimental drug Cabaletta in the U.S. in people with oculopharyngeal muscular dystrophy (OPMD).
The experimental antisense drug will be tested in infants with a genetic spinal muscular atrophy diagnosis who are to date without symptoms
Biogen Idec has announced plans to run a phase 2 clinical study of the experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA)who are less than 6 weeks old and have a genetic diagnosis of the disorder but are not yet showing symptoms.
A drug that has FDA approval for other indications is being tested in CCD patients who are at least 7 years old
The National Institute of Nursing Research (NINR) at the National Institutes of Health in Bethesda, Md., is conducting a study to determine if an antioxidant drug that has been approved by the U.S. Food and Drug Administration for other indications can reduce the severity of some symptoms in people with central core disease (CCD), a genetic disease caused by defects in a structure known as...