Added by PPMD on October 2, 2015 at 10:30am — No Comments
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…Continue
Added by PPMD on September 30, 2015 at 12:00pm — No Comments
The neuromuscular group at Great Ormond Street Hospital, London UK, have recently published their study comparing the effectiveness of echocardiogram with cardiac MRI in…Continue
Added by Kathi Kinnett on September 29, 2015 at 3:00pm — No Comments
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…Continue
Added by Ryan Fischer on September 29, 2015 at 11:08am — No Comments
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…Continue
Added by Pat Furlong on September 28, 2015 at 10:00am — No Comments
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…
Added by Kathi Kinnett on September 25, 2015 at 3:34pm — No Comments
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…Continue
Added by Ryan Fischer on September 25, 2015 at 9:30am — No Comments
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
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…Continue
Added by Ryan Fischer on September 18, 2015 at 10:29am — No Comments
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…Continue
Added by Annie Kennedy on September 16, 2015 at 11:23am — No Comments
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 “…Continue
Added by John D. Porter, PhD on September 16, 2015 at 11:00am — No Comments
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…Continue
Added by Annie Kennedy on September 16, 2015 at 11:00am — No Comments
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…Continue
Added by PPMD on September 15, 2015 at 5:10pm — No Comments
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…Continue
Added by Annie Kennedy on September 14, 2015 at 12:32pm — No Comments
In an effort to provide regular communication on BioMarin's Duchenne muscular dystrophy programs to the Duchenne community, the company is circulating a quarterly update. This update is the first of its kind for BioMarin, and they welcome your feedback to this new communication channel, which you may do by…Continue
Added by PPMD on August 17, 2015 at 12:30pm — No Comments
Summit Therapeutics plc announced that its Phase 1b modified diet clinical trial of SMT C1100 for the treatment of Duchenne met its primary objective with half of the patients who received the higher dose of SMT C1100 achieving desired plasma levels while following specific dietary guidance. Based on these results, Summit will advance SMT C1100 into a Phase 2 open-label clinical trial. Congratulations to our friends at Summit and we look forward to hearing more about next…Continue
Last week, Marathon Pharmaceuticals announced that the company will begin the New Drug Application (NDA) process for deflazacort as a potential treatment for patients…Continue
Added by PPMD on August 12, 2015 at 4:07pm — No Comments
(pictured: DuchenneConnect's Family Advisors at PPMD's 2015 Connect Conference)
DuchenneConnect, led by PPMD, has been approved for…
Added by Ann Lucas on August 12, 2015 at 12:04pm — No Comments
Ad Comm. It’s a term our community will be hearing a lot this fall as companies get closer to the possible approval of treatments for Duchenne. But what is an Ad Comm and how does it involve and affect you or your…Continue
Added by Ryan Fischer on August 11, 2015 at 12:00pm — No Comments
Providers care for many people, with many diagnoses – far too many for any one health care provider to be an expert in them all. Any busy provider would find it difficult to have the time to look over a two-volume document like the Care Considerations [PDF], or even a several-page document like the…Continue
Added by Kathi Kinnett on August 11, 2015 at 9:30am — No Comments