Gene therapy proposes a promising approach in treating Duchenne, placing corrected genes into cells that have missing or damaged genes. Although gene therapy is still relatively early, CureDuchenne is encouraged that Bamboo Therapeutics is planning a human clinical trial for 2017.  As always, we need to be mindful of the difficulty of delivering leading edge therapeutics, but the prospect of having a therapy that treats all Duchenne patients, regardless of mutation, and one that would not require repeat dosing, is very attractive.

Bamboo Therapeutics, along with CureDuchenne, will host a webinar “Bamboo Therapeutics – Developing a novel gene therapy for patients with Duchenne Muscular Dystrophy” on June 13 to explain its gene therapy for Duchenne. The webinar will include Sharon Hesterlee, Ph.D., EVP Patient Advocacy and Public Affairs and Jude Samulski, Ph.D., Scientific Founder, both from Bamboo Therapeutics, as well as Jak Knowles, MD, Vice President Medical and Scientific Affairs and Debra Miller, Founder and CEO, both from CureDuchenne.

Thanks to Bamboo Therapeutics’ work on a gene therapy, which utilizes a disabled virus to bring an improved version of dystrophin to the skeletal and cardiac muscles affected by Duchenne, we are moving in a positive direction. We have been impressed with Bamboo’s team and its approach.  We are proud to have provided funding for Bamboo to help advance their gene therapy treatment for Duchenne.

Our mission is stated in our name: to CureDuchenne. In order to achieve that, we must continue to identify promising research that targets the disease as a whole. The mutation on the X chromosome in individuals with Duchenne results in a partial or complete inability to produce the muscle protein dystrophin, causing muscle deterioration among a variety of other complications. Because Duchenne affects the entire body, treatment requires a multifaceted approach.

As research in exon skipping advances, we must also continue to identify promising research that targets the whole disease. Because heart failure is the leading cause of death among individuals with Duchenne, CureDuchenne has invested in cardiac therapy research as well as therapies to target inflammation, fibrosis and to upregulate or downregulate other proteins that will improve the outcome for Duchenne.  All of these therapies combined, could bring us closer to treating Duchenne as a whole.

Please join us for the webinar on June 13 at 3 p.m. ET/noon PT to learn more.  Register here. The call in number is 844-337-8088 and the conference ID number is 22853541. We would appreciate you sharing information about the webinar with others in the Duchenne community or others who could benefit from this information.

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