PPMD's Blog (474)

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…


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…


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…


Added by PPMD on April 16, 2018 at 12:41pm — 2 Comments

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.…


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

[WEBINAR] What is a Master Protocol?

PPMD fights to end Duchenne for every single family. As part of our mission, we believe that we must continually work to identify and explore innovative opportunities that may accelerate the drug development and clinical trial process, getting effective therapies to individuals with Duchenne as quickly as possible.

Many of the families in our…


Added by PPMD on March 26, 2018 at 12:00pm — No Comments

Santhera Shares Duchenne Community Update on SIDEROS Trial

Santhera has provided a community update, including the latest news on their SIDEROS trial, currently recruiting. Click here to view the update.…


Added by PPMD on March 23, 2018 at 10:02am — No Comments

Sarepta Therapeutics Announces Launch of Route 79, The Duchenne Scholarship Program

Sarepta Therapeutics announced the launch of Route 79, The Duchenne Scholarship Program, an annual scholarship for students diagnosed with Duchenne. The Route 79 program is designed to help students with Duchenne pursue their post-secondary educational goals. Scholarships of up to $10,000 will be awarded to 10 individuals chosen by an independent committee of Duchenne community members based on an applicant’s…


Added by PPMD on March 20, 2018 at 10:54am — No Comments

Solid Biosciences Announces Clinical Hold On SGT-001 Phase I/II Clinical Trial For Duchenne

Solid Biosciences announced that the FDA has placed their Phase I/II clinical trial for SGT-001, IGNITE DMD, on Clinical Hold following a serious adverse event that occurred in the first patient dosed, a non-ambulatory adolescent. The patient was admitted to the hospital, received treatment, and is home with his…


Added by PPMD on March 15, 2018 at 8:30am — No Comments

Roche Shares Update on RG62026 Phase 2/3 Study

Roche has provided an update to the community regarding the paused recruitment of RG6206, an investigational anti-myostatin adnectin, in ambulatory boys with Duchenne. The company hopes to restart recruitment in the second quarter of this year.

Read the update from Roche: …


Added by PPMD on March 14, 2018 at 11:00am — No Comments

Summit Announces New Analysis Showing Ezutromid Significantly Reduced Muscle Inflammation in Phase 2 Clinical Trial in DMD

Summit has announced additional positive data from its PhaseOut DMD clinical trial today, showing a significant reduction in muscle inflammation after 24 weeks of ezutromid dosing. The findings in their 24-week interim data are consistent with the expected…


Added by PPMD on February 26, 2018 at 10:30am — No Comments

New Published Study Reports Cardiosphere-Derived Cells Improved Skeletal and Cardiac Muscle Function in Mouse Model of Duchenne

Capricor Therapeutics announced the online publication in Stem Cell Reports of a new study by researchers at the Smidt Heart Institute at Cedars-Sinai Medical Center, who found that cardiosphere-derived cells (CDCs) improved cardiac muscle function, walking abilities and survival in a mouse model of Duchenne. The CDCs used in the study are the research grade version of CAP-1002,…


Added by PPMD on February 22, 2018 at 2:40pm — No Comments

PPMD to Host Landmark Duchenne Patient-Focused Compass Meeting

Join us in person or virtually!

As part of this year's Advocacy Conference (March 4-6, 2018), PPMD will host the Duchenne…


Added by PPMD on February 21, 2018 at 1:30pm — No Comments

PTC Therapeutics Receives Formal Dispute Resolution Request Decision from the FDA's Office of New Drugs

PTC Therapeutics, Inc. announced that the Office of New Drugs of the U.S. Food and Drug Administration has reiterated the FDA's prior position and denied PTC's appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for ataluren. In its letter, the Office of New Drugs recommended a possible path forward for the ataluren NDA submission…


Added by PPMD on February 20, 2018 at 9:00am — No Comments

[Webinar Recording & FAQs] Positive Data from Summit's PhaseOUT DMD Ezutromid Clinical Trial

Last week, Parent Project Muscular Dystrophy and Action Duchenne hosted a webinar update on the recent report that Summit has announced positive 24-week interim results from the open-label Phase 2 proof of…


Added by PPMD on February 15, 2018 at 11:30am — No Comments

FDA Publishes Finalized Duchenne Guidance for Drug Development

Today is a historic moment for the Duchenne community, as moments ago the FDA published the finalized “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment” Guidance for Industry…


Added by PPMD on February 15, 2018 at 10:30am — No Comments

Catabasis Reports Edasalonexent Preserved Muscle Function & Substantially Slowed Duchenne Progression

Catabasis Pharmaceuticals reported new positive efficacy and safety results showing preservation of muscle function and sustained disease-modifying effects in boys with Duchene in the MoveDMD trial open-label extension following 48 and 60 weeks of treatment with edasalonexent. Consistent improvements in all assessments of muscle function were observed after more than a year of oral 100 mg/kg/day edasalonexent treatment compared to the rates of change in the…


Added by PPMD on February 13, 2018 at 9:00am — No Comments

Santhera Launches U.S. Expanded Access Program with Idebenone for People with Duchenne

Santhera Pharmaceuticals announced the launch of a U.S. Expanded Access Program (EAP) referred to as BreatheDMD with idebenone for people with Duchenne. Through the BreatheDMD program, eligible patients in the U.S. with Duchenne who are 10 years and older and in respiratory function decline, can obtain access to…


Added by PPMD on February 6, 2018 at 8:00am — No Comments

Summit Therapeutics Shares Interim 24-Week Data from PhaseOut DMD Clinical Trial

Today, Summit announced positive 24-week interim data from PhaseOut DMD, their Phase 2 clinical trial of the utrophin modulator ezutromid.…


Added by PPMD on January 25, 2018 at 10:00am — No Comments

We did it! Because of your commitment & courage!

Parent Project Muscular Dystrophy believes in the promise that gene therapy holds for the Duchenne community. And so we set an ambitious fundraising goal for our Gene Therapy Initiative. In fact, $500,000 is our largest fundraising goal ever.

And once…


Added by PPMD on January 3, 2018 at 9:30am — No Comments

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