Guangping Gao, PhD, will be honored by the National Tay-Sachs & Allied Diseases Association for his work in identifying the Canavan gene, revitalizing gene therapy, and his many accomplishments leading to potential treatments for Canavan disease.
/PRNewswire/ CureDuchenne, a leading global nonprofit focused on finding and funding a cure for Duchenne muscular dystrophy (DMD), today released a webinar.
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The Treatabolome project is a research initiative to develop a freely available, interoperable online platform to disseminate rare disease and gene-specific treatment information to healthcare professionals regardless of their level of specialized expertise to reduce treatment delays. This initiative is highly relevant to neuromuscular disorders as they are rare diseases by definition. In this special issue of the Journal of Neuromuscular Diseases, experts contribute Treatabolome-feeding systematic literature reviews on rare neurological and neuromuscular disorders.
The Treatabolome project is a research initiative to develop a freely available, interoperable online platform dedicated to disseminating rare disease and gene-specific treatment information to healthcare professionals regardless of their level of specialized expertise.