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All Blog Posts Tagged 'Staff' (567)


Staff
FDA Releases New Date for Eteplirsen Ad Comm

At long last we have received information regarding the rescheduled Advisory Committee for Sarepta (eteplirsen). The Ad Comm will take place on Monday, April 25 at the College…

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Added by PPMD on March 10, 2016 at 9:00am — No Comments


Staff
How You Can Participate in Upcoming FDA Advisory Committee Meetings

UPCOMING AD COMMS

Sarepta…

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Added by PPMD on March 10, 2016 at 9:00am — No Comments


Staff
A Win in Washington Today – For Every Single Person Living with Duchenne

Our Duchenne community is on our way to achieving ANOTHER milestone.

 

MD-CARE Act passed 2001

MD-CARE Act reauthorized 2008

MD-CARE Act Amended 2014

Heavy Engagement within PDUFA V; FDASIA reauthorization 2012

Today,…

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Added by Annie Kennedy on March 9, 2016 at 11:30am — No Comments


Staff
Gene Therapy Today: Notes from PPMD’s Duchenne Gene Therapy Forum



Gene therapy.

Those two words have been tossed around for some time, starting in the late 1990s when several gene therapy trials started, but ended with fatal results. Now, when the field has learned a lot from those initial results and with optimized technologies, we come back to gene…

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Added by Abby Bronson on March 8, 2016 at 12:00pm — 4 Comments


Staff
PPMD Launches the Every Single [One] Tour

For 22 years, PPMD has brought together families with leading experts, care providers, and industry partners through our Annual Connect Conference,…

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Added by PPMD on March 4, 2016 at 10:00am — No Comments


Staff
Registration for PPMD's 2016 Connect Conference is Now Open

Parent Project Muscular Dystrophy's Annual Connect Conference is a unique convergence of industry partners, scientific leaders, medical providers, people…

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Added by PPMD on March 1, 2016 at 9:30am — No Comments


Staff
DuchenneConnect – More Than Clinical Trial Updates

 

Did you know that even if you are not interested in being in a clinical trial, DuchenneConnect is still a powerful tool that is useful for people with Duchenne and Becker? That’s right! DuchenneConnect is the largest, most robust patient report registry for Duchenne and Becker in the United…

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Added by Ann Lucas on February 29, 2016 at 8:30am — 3 Comments


Staff
PPMD Partners with AAP & CDC to Develop New Tool to Help Parents Detect Motor Development Delays

PPMD is proud to have been chosen by American Academy of Pediatrics (AAP) and the Centers for Disease Control and Prevention (CDC) to help create a new interactive tool,…

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Added by PPMD on February 24, 2016 at 10:00am — No Comments


Staff
ACTION ALERT: Urge Lawmakers to Keep Driving Progress in the Fight to End Duchenne

Urge your Lawmakers to Sign the FY 17 Duchenne Appropriations Letter!

 

Today and tomorrow, scores of PPMD advocates will be on Capitol Hill to urge their Senators and Members of Congress to…

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Added by Ryan Fischer on February 22, 2016 at 1:43pm — No Comments


Staff
Capricor Therapeutics Announces the First Patient with Duchenne-Related Cardiomyopathy Treated with CAP-1002 in the HOPE-Duchenne Phase I/II Clinical Trial

Capricor Therapeutics announced continuing enrollment of, and the treatment of the first patient with Duchenne-related cardiomyopathy with CAP-1002 in the HOPE-Duchenne Phase I/II clinical trial. This is an exciting next step in the  progress of CAP-1002, an allogeneic, cardiosphere-derived stem cell (CDC) therapy.



As you may recall, PPMD hosted a webinar with Capricor last month. Click below to watch the webinar,…

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Added by PPMD on February 22, 2016 at 10:30am — No Comments


Staff
Finding Routes to Duchenne Drug Approvals

It seems that everyone is looking for the newest molecule, the newest therapy, the newest technique that will halt the progression of Duchenne. This is good and we should continue on this journey. But none of these novel treatments will get to patients without first going through a rigorous…

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Added by Abby Bronson on February 20, 2016 at 10:28am — No Comments


Staff
Recognizing the Symptoms of Fat Embolism Syndrome (FES)

It is a sadly familiar story: the parent moves the child, the child gets bumped/dropped, the child complains that something hurts (but doesn't really hurt so badly that a broken bone is suspected), and everyone goes on about their business.

Then, the child may become sleepier than…

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Added by Kathi Kinnett on February 19, 2016 at 10:00am — No Comments


Staff
Congress Calls on FDA to Increase Efforts to Treat Duchenne



Thanks to this incredible community urging lawmakers, well over 100 Members of Congress signed and submitted a letter …

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Added by Ryan Fischer on February 18, 2016 at 2:40pm — 1 Comment


Staff
Duchenne Dental Dilemmas: Bisphosphonates & Osteonecrosis of the Jaw

Medication-related osteonecrosis of the jaw (MRONJ),  or osteonecrosis of the jaw (ONJ), is a condition that results when the bones of the jaw are exposed and then do not heal  generally when a tooth is removed.…

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Added by Kathi Kinnett on February 11, 2016 at 11:30am — No Comments


Staff
PPMD Announces Gene Therapy Forum

This year’s already action-packed Advocacy Conference, February 21-23 welcomes a special Gene Therapy Forum on Monday, February…

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Added by Abby Bronson on February 10, 2016 at 10:30am — 2 Comments


Staff
Update on Summit Clinical Trials

Summit Therapeutics’ SMT C1100 is a small molecule drug that targets increasing the production of utrophin. Utrophin is a protein that is present in early muscle development but “switched off” as muscle fibers mature and dystrophin is…

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Added by Ann Martin on February 9, 2016 at 1:00pm — No Comments


Staff
PDUFA Extension for the FDA's Review of Eteplirsen

Sarepta Therapeutics received notification of a PDUFA extension for the FDA's review of eteplirsen. The PDUFA date was extended by an…

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Added by PPMD on February 8, 2016 at 10:00am — No Comments


Staff
ACTION ALERT: Sign-On Extended! Urge House Member - Apply FDASIA Tools to Duchenne

Action Alert

PPMD and a coalition of Duchenne foundations continue to work together on preparing for the Sarepta Ad Comm. As of today, we are still waiting on a new confirmed date. With the postponement of the Ad Comm, the Congressional letter to FDA remains open. Has your House Members signed on…

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Added by Ryan Fischer on January 25, 2016 at 11:30am — No Comments


Staff
FDA Ad Comm for Eteplirsen Postponed Due to Inclement Weather

We are deeply saddened, disappointed, and distressed to report that the highly-anticipated Ad Comm for eteplirsen has been postponed due to the blizzard anticipated to hit DC on Friday. We are grateful to our extraordinarily powerful Duchenne community who has been willing to travel into the eye of this storm to fight Duchenne. Our community has proven once again to be a force stronger than any winter storm, and much stronger than Duchenne itself. As soon as we receive any updates…

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Added by PPMD on January 20, 2016 at 7:09pm — No Comments


Staff
ACTION ALERT: Urge House Member - Apply FDASIA Tools to Duchenne

There has never been a moment like this in our Duchenne community. Today the FDA released briefing documents on the application for eteplirsen. It is clear that we continue to face a serious regulatory challenge as a community.…

Action Alert

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Added by Ryan Fischer on January 15, 2016 at 2:45pm — 1 Comment

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