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…

PPMD and NIH are convening a group of more than 60 researchers, post-docs, neurologists, surgeons, industry partners, physiatrists, and physical therapists on April 19-20, 2018, who have experience and expertise in the development, prevention, and management of contractures to discuss these critical issues. 

We just don’t know very much about…

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…

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…

We are pleased to report our final numbers for the FY19 letters.

In total, 19 Senators signed onto the Senate FY19 letter, joining 51 members of the House. That makes a solid 70 members of Congress (House and Senate combined) sending a strong message of support behind Duchenne research, public health, drug development, and patient support initiatives. They provide strategic direction for the federal agencies that touch Duchenne. Given the…

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.…

Just like a house needs a stable frame, your body needs a stable skeleton. Bone health is important for anyone living with Duchenne, whether walking (ambulatory) or those no longer able to walk without the assistance of a wheelchair or scooter (non-ambulatory). People of all ages living with Duchenne have weak bones, especially if they are taking steroids…