September 2015 Blog Posts (15)

PPMD Launches Next Phase of Genetic Testing Program, Decode Duchenne in collaboration with BioMarin Pharmaceutical, PTC Therapeutics, & Sarepta Therapeutics

PPMD is excited to today announce a collaboration with BioMarin Pharmaceutical Inc., PTC Therapeutics, and Sarepta Therapeutics, Inc. in the next phase of the highly successful Decode Duchenne program. Decode Duchenne is a nationwide program to assist individuals with…


Added by PPMD on September 30, 2015 at 12:00pm — No Comments

New Publication Demonstrates Improved Value of Cardiac MRI in Duchenne Muscular Dystrophy

The neuromuscular group at Great Ormond Street Hospital, London UK, have recently published their study comparing the effectiveness of echocardiogram with cardiac MRI in the assessment Duchenne cardiomyopathy in patients preparing for surgical…


Added by Kathi Kinnett on September 29, 2015 at 3:00pm — No Comments

The Ensuring Access to Clinical Trials Act (EACT) Passes Senate, then House

We are thrilled to report that last night the Ensuring Access to Clinical Trials Act of 2015 (EACT) passed a vote in the House of Representatives (after passing the Senate by unanimous consent in July) and will go to the President for his signature!

This a result of everyone who contacted their Representative – over 400 families in…


Added by Ryan Fischer on September 29, 2015 at 11:08am — No Comments

“Hullabaloo in Duchenne”

Friday was one of those days, meetings in Washington all week and anxious to get home. Traffic was a mess, Friday evening flights out of DC chaotic. I worried I would miss my flight, the last one to Cincinnati. I attempted to guesstimate the amount of time to get through the security line, grab a salad, and still make the flight. I made it just in time to learn my…


Added by Pat Furlong on September 28, 2015 at 10:00am — No Comments

Update of the Duchenne Care Considerations

Right now, Annie Kennedy and I are sitting in a room, in Atlanta at the Centers for Disease Control and Prevention (CDC), with a group of experts in Duchenne from around the world. It is the culmination of 2-year effort and we are delighted that…


Added by Kathi Kinnett on September 25, 2015 at 3:34pm — No Comments

ACTION ALERT: Don't Let Critical Senate Legislation Stall!

Urge your Representative to Pass the Ensuring Access to Clinical Trials Act of 2015 (H.R. 209)


One minute of your day could mean millions of minutes for those diagnosed with Duchenne.

Today, patient advocates from across the country will unite in a day of action to urge the Senate to make medical innovation a top priority.

Earlier this year the Duchenne…


Added by Ryan Fischer on September 25, 2015 at 9:30am — No Comments

FDA Advisory Committee Meetings: An Overview of the Process & Ways to be Involved

On September 2nd, PPMD convened a public webinar for the Duchenne community focused on FDA Advisory Committee Meetings (or “Ad Comms”). After working closely with representatives from FDA, we are excited to share some of the frequently asked questions that arise regarding the Ad Comm process, as well as some specifically relevant to our community. As we get closer to possible dates for these meetings, we will add information as it becomes available.…


Added by PPMD on September 24, 2015 at 3:30pm — No Comments


There are days when Duchenne gets the best of me. Today is one of those days.

The feeling doesn’t happen often, thankfully. If it did, I’d probably go crazy, as would…


Added by Dave Click on September 22, 2015 at 11:30pm — No Comments

ACTION ALERT: Urge your Representative to Pass the Ensuring Access to Clinical Trials Act of 2015 (H.R. 209)

As we make advancements in Duchenne drug development, the potential therapeutic opportunities for Duchenne patients across the spectrum of the disease continues to grow — as do the opportunities to participate in clinical trials. This is a hopeful and exciting time for our Duchenne community and we must ensure unnecessary barriers don’t hamper clinical…


Added by Ryan Fischer on September 18, 2015 at 10:29am — No Comments

Meet Our Newest PPMD Adult Advisory Committee Member: Jen Petrusko Bauer, manifesting carrier

There are moments in life that take your breath away. When you are truly in awe of the power and grace of others.


About a year ago, we put our a call to our community asking for adults living with Duchenne and Becker to consider applying to be a part of our Innagural PPMD Adult Advisory Committee (PAAC). We were overwhelmed by the response we received and were thrilled by the…


Added by Annie Kennedy on September 16, 2015 at 11:23am — No Comments

The Power is in Your Hands: Duchenne Regulatory Science Consortium

About a month ago, I wrote to you to announce the launch of a new partnership between PPMD and the Critical Path Institute (C-Path) called the Duchenne Regulatory Sciences Consortium or D-RSC (see “Different Together: PPMD Announces the…


Added by John D. Porter, PhD on September 16, 2015 at 11:00am — No Comments

Join the 2016 PPMD Adult Advisory Committee – Apply by October 23!

We’re Looking for a Few (More) Good Men!

A year ago, we had a vision that we would begin to formalize the adult voice of our Duchenne community by convening a leadership group comprised of young adults and adults of accomplished and passionate men who also had Duchenne and…


Added by Annie Kennedy on September 16, 2015 at 11:00am — No Comments

Bennet, Burr, Warren, Hatch Introduce Bill to Help Patients by Facilitating Faster Development of Drugs for Rare Diseases

We are thrilled to announce that the Advancing Targeted Drugs for Rare Diseases Act bill dropped in the Senate today (see press release below)! PPMD has been working with the Senate HELP Committee and Sarepta for the last several months on this 21st Century Cures provision which morphed into stand-alone legislation (view PPMD's most updated endorsement of this…


Added by PPMD on September 15, 2015 at 5:10pm — No Comments

Webinar Recording: How FDA Ad Comms Work & What Role You Can Play

Ad Comms – What’s the buzz all about? If you’ve been anywhere near social media lately, you’ve likely seen the term ‘Ad Comm’ referenced. Speculation about dates, implications for approvals and access to therapies we all care about, questions about…


Added by Annie Kennedy on September 14, 2015 at 12:32pm — No Comments

PPMD Receives $30,000 STRIVE Award From PTC Therapeutics

On the eve of the 2nd Annual World Duchenne Awareness Day, PPMD is excited to be one of five recipients of PTC Therapeutics’ STRIVE Awards! This $30,000 grant will be used to help PPMD launch a campaign for…


Added by PPMD on September 4, 2015 at 8:46am — No Comments

Monthly Archives















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



© 2021   Created by PPMD.   Powered by

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