Connect with us



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.

Newsletter Signup

Staff reporter
Written By


sight sight

Blindness cure in sight


Opening the door to healthy DNA


Gene therapy breakthrough


Gene therapy could prevent blindness


Newsletter Signup