Michael Huang, the NHMRC Peter Doherty Postdoctoral Fellow in the department of pathology, Bosch Institute, at the University of Sydney, Australia, was awarded an MDA development grant totaling $177,100 over a period of three years to explore how deficiency of the frataxin protein in Friedreich’s ataxia (FA) may alter the function of cellular power supplies . . .
The investigational drug amifampridine phosphate (brand name Firdapse), under development by Catalyst Pharmaceuticals, has received U.S. Food and Drug Administration (FDA) Orphan Drug designation for the treatment of myasthenia gravis (MG). Amifampridine phosphate is a potassium channel inhibitor designed to prolong signals released from nerves and allow greater stimulation of muscles. The drug currently is . . .
Researchers at the National Institutes of Health (NIH) seek children with neuromuscular disorders (NMDs) and/or their caregivers (parent or legal guardian) to complete questionnaires. Researchers would like to develop a tool to better assess the quality of life of people with NMDs, such as muscular dystrophy. The goal of this study is to validate two different . . .
Today we couldn’t be happier as, together with the Duchenne community, we celebrate FDA accelerated approval of eteplirsen to treat kids and adults with some forms of Duchenne muscular dystrophy (DMD). “This is something that we’ve watched, and hoped and prayed for, and seen it develop over the last seven years or so,” said Josh . . .
Henry Houlden, professor of neurology at the MRC Centre for Neuromuscular Diseases, University College London Institute of Neurology in England, was awarded an MDA research grant totaling $288,151 over three years to elucidate the genetic causes of severe forms of Charcot-Marie-Tooth disease (CMT) and other types of early-onset neuropathy. Identification of the causative genes could . . .
Rachelle Crosbie-Watson, professor and vice-chair of graduate education at the University of California Los Angeles, was awarded an MDA research grant this summer totaling $300,000 over a period of three years to test new therapies for cardiomyopathy (heart muscle weakness) associated with Duchenne muscular dystrophy (DMD). Can you tell us a little bit about your . . .
Since its inception, MDA has invested more than $1 billion in neuromuscular disease research to uncover new treatments and cures. In 2016 alone, MDA awarded 66 new research, development and research infrastructure grants with a total funding commitment of more than $17 million. These and other MDA grants fund research projects designed to uncover the . . .
Steven Markus, assistant professor at Colorado State University in Fort Collins, Colo., was awarded an MDA research grant totaling $300,000 over a period of three years to study alterations in the dynein gene and their effects in spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS). His work to elucidate the molecular basis for dynein . . .
Natalia Rodríguez Muela, a postdoctoral fellow at Harvard University in Cambridge, Mass., was awarded an MDA development grant totaling $179,985 over a period of three years to deepen our understanding of what goes wrong in spinal muscular atrophy (SMA), a progressive disease caused by low levels of SMN protein. The work will focus on how . . .
Investigators are seeking people to participate in a phase 2 clinical trial, sponsored by Idera Pharmaceuticals, to evaluate the effect of the experimental drug IMO-8400 on skin lesions and muscle weakness in dermatomyositis. While the cause of dermatomyositis is unclear in most cases, it is known that ongoing inflammation triggered by the disease, over time, . . .
