PPMD's Blog – February 2018 Archive (8)

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

Monthly Archives










Need help using this community site? Visit Ning's Help Page.



© 2022   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service