PPMD's Blog – April 2018 Archive (4)

Capricor Announces New Pre-Clinical Study Finds Repeat Doses of CAP-1002 Lead to Enhanced Exercise Capacity in Duchenne Muscular Dystrophy Disease Model

Capricor Therapeutics has announced that researchers found that repeat dosing of the company’s proprietary cellular therapy, CAP-1002, yields an increase in exercise performance in a disease model of Duchenne muscular dystrophy, the mdx mouse. The upcoming HOPE-2 clinical trial will test the safety and efficacy of repeat doses of CAP-1002 in boys and young men with…

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Added by PPMD on April 20, 2018 at 9:30am — 1 Comment

Solid Biosciences Provides Update On SGT-001 Clinical Development Program For Duchenne

Solid has provided a letter to the community with the latest news on their halted IGNITE DMD program due to a serious adverse event experienced by the first patient dosed with SGT-001. While the patient continues to do well, Solid has been able to successfully resolve the previously-announced partial clinical hold on the planned high dose of SGT-001 in IGNITE DMD, which was due to manufacturing-related questions from the FDA. While encouraging, IGNITE DMD remains on hold until they…

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Added by PPMD on April 18, 2018 at 8:30am — No Comments

Pfizer Doses First Patient Using Investigational Mini-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy

PPMD is thrilled to learn that Pfizer Inc. has initiated a Phase 1b clinical trial for its mini-dystrophin gene therapy candidate, PF-06939926, in Duchenne, with the first boy receiving an infusion of the mini-dystrophin gene on March 22, administered under the supervision of principal investigator, Edward Smith, MD, Associate Professor of Pediatrics and Neurology at Duke University Medical Center. Screening and enrollment of patients is expected to continue at up to four clinical…

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Added by PPMD on April 16, 2018 at 12:41pm — 1 Comment

Roche Restarts RG62026 Phase 2/3 Study

Roche has provided an update to the community announcing that they have restarted recruitment of RG6206, an investigational anti-myostatin adnectin, in ambulatory boys with Duchenne. PPMD will be hosting a webinar with Roche in the coming weeks and we look forward to learning more.…

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Added by PPMD on April 11, 2018 at 4:30pm — No Comments

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