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…

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…

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…

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,…

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…

URGE THE SENATE TO PRIORITIZE CURES!

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…

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.…

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

The feeling doesn’t happen…

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…

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…

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 “…

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…

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…

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…

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…