February 2016 Blog Posts (16)

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…


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

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, "Physical Developmental Delays: What to Look For" that we…


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

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 continue supporting vital efforts that will move us closer to ending…


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

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,…


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

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 evaluation in clinical trials.



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



Added by Dave Click on February 19, 2016 at 2:30pm — 1 Comment

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 usual, may start to act a little odd, and slowly develops…


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

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 …


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

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. It has been stated that bisphosphonates (medications that…


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

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 22 from 9am to 1pm at The Mayflower Hotel in D.C. Hosted…


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

Advancing Targeted Therapies for Rare Diseases Act Goes Before HELP Committee

For nearly two years, PPMD has been working with Congressional champions and industry partners to help lead an effort to streamline the regulatory pathways and review processes for targeted therapeutics (which would include such things as ‘follow on exons’) by clarifying the FDA’s existing authority to leverage data previously used in the approval of a targeted product when approving a new therapy. These efforts have yielded a provision in the House of Representatives…


Added by Annie Kennedy on February 9, 2016 at 3:36pm — 8 Comments

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 produced. SMT C1100 hopes to maintain the production of utrophin,…


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

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 extension period of three months to May 26, 2016. While this…


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

Lilly Trial Finishes With Disappointing Results

Lilly has now completed one of the largest clinical trials in Duchenne (330 patients) with disappointing results. Today the company reports that their Phase 3 study of tadalafil failed to provide evidence for efficacy in slowing decline in walking ability in boys with Duchenne. Subsequently, the company has made the decision to discontinue the open-label extension. The full update to the community is below.


Sadly, this is one more blow for this community. We are deeply…


Added by PPMD on February 3, 2016 at 8:53am — 1 Comment

Masters Update: New Tracking System

The UK has instituted new regulations around shipping medications out of the country.  The UK is now requiring companies to provide proof of drugs that they have exported overseas.  In order to be compliant, Masters Marketing will begin to ship deflazacort, and other drugs, using trackable service providers.  Using this tracking system, Masters will be able to track orders and reduce the number of lost shipments.    


When you order medications,…


Added by PPMD on February 1, 2016 at 3:58pm — No Comments

Growing Our Team

We are not a team because we work together. We are a team because we respect, trust, and care for each other. – Vala Afshar


I am the first person to admit that I don’t know much about a lot of different things. But I do know talent when I see it. And I could not be more proud of the team of passionate, dedicated leaders…


Added by Pat Furlong on February 1, 2016 at 9:43am — No Comments

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