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Staff
PPMD posted a blog post

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, Capricor’s cell…See More
18 hours ago
Profile IconAnna Stanejko, Nick Wilkins, Ronda M Roda and 1 more joined PPMD Community
yesterday

Staff
PPMD posted a blog post

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 Patient-Focused Compass Meeting: Navigating our Pathway Forward. The meeting will be held on Monday, March 5, 2018 at the Mayflower…See More
yesterday
Margarita Yashchenko favorited MicahsDaddy's group California Families
Wednesday
Margarita Yashchenko shared MicahsDaddy's group on Facebook
Wednesday
ven favorited Debbie Schneider's discussion COQ10 versus Idebenone?
Wednesday

Staff
PPMD posted a blog post

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 based on the accelerated…See More
Tuesday
Julie Caron Burslem is now a member of PPMD Community
Tuesday
Robin Taylor commented on Pat Furlong's blog post First Duchenne Patient Dosed in Microdystrophin Gene Therapy!
"Do we have an update on the young boy who received the first DMD gene therapy?  Keeping him, his caregivers and family in our prayers."
Feb 15

Staff
Ryan Fischer posted a blog post

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 with Michael's Cause, Pietro's…See More
Feb 15
Robin Taylor replied to ven's discussion Brenda Wong Leaving CMC
"Do you know where Dr Wong is going?"
Feb 15
Arlington Babb is now a member of PPMD Community
Feb 15

Staff
PPMD posted blog posts
Feb 15
Keith & Jill VE replied to Keith & Jill VE's discussion Emflaza (aka deflazacort) Approvals
"Actually, quite the opposite!  Last year, PTC was charging my insurance company a ridiculous $6800/MONTH for Emflaza.  As of 2018, the monthly cost has RISEN to almost $7400!   What I've learned is that PTC will cover any…"
Feb 14
Profile IconSilje Therese Brattsti, Vanja Siljeg and Becky Lilley joined PPMD Community
Feb 13

Staff
PPMD posted a blog post

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 pre-specified control period for boys…See More
Feb 13
 
 
 

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