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Progress in fight against rare disease


A leading biosciences company is developing a new gene therapy that shows signs of improving muscle function in patients suffering from a severe form of muscular dystrophy.

Solid Biosciences announced this week that data from its preclinical trials showed the gene therapy, called SGT-001, could be crucial in helping to combat Duchenne muscular dystrophy (DMD), a rare muscle-wasting disease effecting young boys.

Dr Joel Schneider, vice president of Research and Development at Solid Biosciences, said the results – presented at the annual meeting of the American Society of Gene and Cell Therapy – underlined the company’s confidence in its “plan to initiate [its] clinical program later this year” as it worked to bring meaningful treatments to patients with this devastating disease.”

Clinical trials for SGT-001 are due to commence in the second half of 2017.

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