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Duchenne Research: 2017 in Review

December 7, 2017

2017 has been an exciting year for Duchenne muscular dystrophy research! As we get close to the end of the year, we’re looking back at some of the stories that caught our attention. You can find these and other news stories here.

Defeat Duchenne Canada grants more than $1 million internationally to Duchenne researchers In May, the Defeat Duchenne Canada board of directors approved the funding of four new Duchenne research projects, bringing our total funding commitment for 2017 to more than $1 million. These new projects include studies in Ontario, Washington, the UK, and Missouri.Click here to read the full story.

Akashi Therapeutics receives FDA clearance for clinical development Akashi Therapeutics now have the greenlight from the FDA to continue clinical development of HT-100. The biopharmceutical company is developing treatments for patients suffering from Duchenne muscular dystrophy.Click here to read the full story.

PTC Therapeutics completes acquisition of Emflaza™ for the treatment of Duchenne muscular dystrophy in the US PTC Therapeutics, Inc. announced it has completed its acquisition of all rights to Emflaza™ (deflazacort) for the treatment of Duchenne muscular dystrophy in the U.S. Execution of the asset purchase agreement setting forth the terms of the acquisition was announced on March 16, 2017.Click here to read the full story.

Translarna beneficial for patients in transitional stage of nonsense mutation Duchenne, study finds The latest results of the Phase 3 clinical trial for PTC Therapeutics’ candidate Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) showed that it can benefit patients who are in a transition stage of the disease and are able to walk between 300 and 400 meters in six minutes.Click here to read the full story.

Microdystrophin gene therapy shows promise in dogs with Duchenne muscular dystrophy, study shows Injecting a smaller but functional form of the dystrophin gene, called microdystrophin, into dogs naturally affected by Duchenne muscular dystrophy allowed them to recover muscle strength and stabilized their overall disease symptoms, a new study shows.Click here to read the full story.Click here to see video of the dogs in the study.

Sick Kids scientists repair genetic errors in mice with muscular dystrophy Sick Kids scientists wielding a “breakthrough” gene editing technology have snipped out a genetic defect in mice that causes congenital muscular dystrophy, eliminating all signs of paralysis in the animals. Defeat Duchenne Canada is funding a study at Sick Kids to research the benefits of this technique in Duchenne muscular dystrophy.Click here to view the full story.Click here for more information on the study Defeat Duchenne Canada is funding.

FDA rejects new drug application for Translarna to treat Duchenne muscular dystrophy The U.S. Food and Drug Administration decided not to approve Translarna (ataluren), by PTC Therapeutics, as a treatment for specific types of Duchenne muscular dystrophy caused by “nonsense” mutations.Click here for the full story.

Solid biosciences readies clinical trial of SGT-001, a potential Duchenne gene therapy Solid Biosciences announced the pending start of a first clinical trial to test the company’s leading investigational therapy – called SGT-001, a microdystrophin gene transfer candidate, as a potential treatment for Duchenne muscular dystrophy (DMD). Defeat Duchenne Canada granted funding for some of the pre-clinical studies that led to this exciting development.Click here to read the full story.

Capricor set to launch phase 2 trial of cell therapy CAP-1002 in advanced Duchenne patients Capricor Therapeutics has received a green light from the U.S. Food and Drug Administration to launch a Phase 2 study of CAP-1002 cell therapy in children and adults with advanced Duchenne muscular dystrophy.Click here to read the full story.