All Blog Posts Tagged 'PPMD Blog' (819)


Staff
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


Staff
PPMD & NIH to Host “Contractures in Duchenne and Other Neuromuscular Conditions”

 

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…

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Added by Kathi Kinnett on April 19, 2018 at 9:30am — No Comments


Staff
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


Staff
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 — No Comments


Staff
End Duchenne Tour Recap: Grand Rapids, MI

PPMD was excited to head to the Midwest for the second stop on the 2018 End Duchenne Tour. The event took place at Western Michigan University in Grand Rapids, Michigan, in…

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Added by Ryan Fischer on March 28, 2018 at 4:30pm — No Comments


Staff
[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…

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


Staff
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.…

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


Staff
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…

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


Staff
ADD YOUR VOICE: Participate in PPMD’s Gene Therapy Survey



Patient-Focused Drug Development in action



Companies developing gene therapies, the FDA, and other stakeholders are interested in the treatment priorities of the Duchenne community.  Because of this, PPMD has collaborated with RTI…

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Added by Ryan Fischer on March 14, 2018 at 1:30pm — No Comments


Staff
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.…

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


Staff
Duchenne Pulmonary Awareness Video Series

Pulmonary management in Duchenne (all of the care involved with breathing and coughing) can be confusing and difficult to understand. There are many terms, abbreviations, and procedures that all sound very complicated. PPMD – with support from Santhera Pharmaceuticals and input from a panel of parents,…

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Added by Kathi Kinnett on March 13, 2018 at 9:30am — No Comments


Staff
Add Your Voice to the Historic Compass Meeting

There are hardly words to capture the impact our unified community had on Monday. We had traveled from all over the nation to convene with federal policymakers, industry partners, and payers for the Duchenne Patient-Focused Compass Meeting: Navigating our Pathway…

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Added by Pat Furlong on March 8, 2018 at 2:00pm — No Comments


Staff
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…

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


Staff
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,…

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


Staff
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…

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


Staff
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…

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


Staff
End Duchenne Tour Recap: Morristown, NJ

PPMD kicked off the 2018 End Duchenne Tour with a stop in our very own backyard - Goryeb Children's Hospital in Morristown, New Jersey! PPMD was extremely grateful to partner…

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Added by Ryan Fischer on February 15, 2018 at 2:30pm — No Comments


Staff
[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…

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


Staff
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…

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


Staff
Action Alert! Americans with Disabilities Act (ADA) in Jeopardy

The Americans with Disabilities Act (ADA) is in jeopardy and a current bill in the House of Representatives threatens to undermine the protections of the ADA.



We need all members of our Duchenne community to contact your…

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Added by Annie Kennedy on February 12, 2018 at 9:30am — No Comments

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