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limb-girdle muscular dystrophy 2b (lgmd2b) affects movement in the arms, legs, fingers, toes, and face. lgmd2b is known to cause weakness and atrophy in the pelvic and shoulder muscles as well, according to the u.s. department of health and human services .
“currently, patients with lgmd2b have no gene or drug-based therapies available to them, and we are amongst the few centres developing therapeutic approaches for this disease,” jyoti k. jaiswal, phd, senior investigator of the center for genetic medicine research at children’s national said in a statement . “we are working to further enhance the efficacy of this approach and perform a longer-term safety and efficacy study to enable the clinical translation of this therapy.”
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jaiswal says due to the size of the gene mutated in lgmd2b, the entire body has difficulty getting the proteins it needs to build muscle and gene therapies are a significant challenge to develop. researchers developed another way to slow the disease’s progression by expanding on a breakthrough they discovered earlier: that the acid sphingomyelinase (hasm) protein is necessary to rebuild injured muscle cells. that research was published in 2014 in the science journal nature .
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researchers say that clinical trials are required next to examine the therapeutic uses of their new form of treating lgmd2b patients.
