NDF Awards $700,000 in Grants for GNE Myopathy Research and Programs
March 13, 2019
Rare Daily Staff
The Neuromuscular Disease Foundation awarded a total of $700,000 in new grants to support research projects and programs related to GNE Myopathy, an adult onset, rare, genetic, distal muscle disease.
GNE Myopathy (GNEM), also known as Hereditary Inclusion Body Myopathy (HIBM), causes severe muscle weakness in the legs and arms, usually leading to physical disability within twenty years of onset. GNE Myopathy does not affect the vital organs, such as the heart, lungs, or the ability to swallow.
The grants announced by the organization are focused on supporting the development of a gene therapy to treat the condition.
“Finding scientists dedicated to your cause is always a challenge for any rare disease, and NDF has worked hard to foster relationships with preeminent scientists and partners in the GNEM community, worldwide,” said Lale’ Welsh, NDF’s CEO. “Our challenge has been to leverage these much-needed funds to encourage scientific collaboration, build on one another’s work with a unified vision, and expedite a therapy that is much too slow for most patients—in spite of the rapid speed of technological progress.”
Recipients include Hadassah Medical Center, Yale University, and the National Institutes of Health.
Since raising the first $1 million to meet a $2.5 million match grant, the NDF has invited numerous proposals for GNEM clinical research projects. The majority of studies directly support a gene therapy. The organization said some remaining proposals are still under review and the organization plans to work with applicants to modify them to enhance their relevance to NDF’s scientific objectives.
NDF said it has also earmarked funds for its other programs that support patient advocacy and awareness.
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