The Muscular Dystrophy Association (MDA) has announced a new round of grant funding that includes second-generation precision medicine treatments, disease-specific research networks, analysis of large data sets, and continued studies on the molecular mechanisms of neuromuscular disease.
The MDA is also expanding the data registry that it started at a subset of the 150 MDA care centers in the US, becoming part of the MOVR (neuroMuscular ObserVational Research) data hub where medical, genetic, and patient-reported data from patients with a variety of neuromuscular diseases will be collected. It is anticipated that MOVR will provide important insights into neuromuscular disease and enable the identification of appropriate patients for clinical trials and improve clinical care.
Grace Pavlath, PhD , MDA Senior Vice President and Scientific Director noted, “MDA is fully committed to accelerating therapies and cures for neuromuscular disease by funding innovative science throughout the drug development spectrum.”
Second-generation treatment research funded includes the development of nanoparticles for delivery of gene-based therapies, which may be more accurate and effective than the adeno-associated virus (AAV) method currently in use in gene therapy trials today.
Continued molecular disease research funded also includes antisense oligonucleotide therapy for spinal-bulbar muscular atrophy (SBMA), the role of histone deacetylase inhibitors in amyotrophic lateral sclerosis (ALS), and identifying novel gene mutations in congenital muscular dystrophy (CMD) and in congenital myopathy (CMY) using advanced genetic techniques.
Grants that will increase collaboration among MDA care centers include funding of 2 disease-specific research networks, for facioscapulohumeral muscular dystrophy (FSHD) and limb-girdle muscular dystrophy (LGMD). Creation of these disease-specific networks is expected to accelerate progress in clinical trials for these diseases. A research infrastructure grant to develop computational tools that can integrate and analyze complex data sets from patients with ALS was also funded.Next Story