PPMD Launches Next Phase of Genetic Testing Program, Decode Duchenne in collaboration with BioMarin Pharmaceutical, PTC Therapeutics, & Sarepta Therapeutics
Thanks to the collaboration and generous support of these partners, Decode Duchenne will continue to provide genetic testing, interpretation, and counseling at no cost to eligible patients who are unable to access testing due to barriers such as a lack of insurance or insufficient insurance coverage.
New Publication Demonstrates Improved Value of Cardiac MRI in Duchenne
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 procedures.
The Ensuring Access to Clinical Trials Act (EACT) Passes Senate, then House
We are thrilled to report that 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 including over 400 families in this community alone!
“Hullabaloo in Duchenne”
PPMD believes in treatments for ALL. We believe that all safe and effective treatments deserve quick approval. We believe that patients, that our loved ones living with Duchenne, deserve options. Maybe you like Tylenol over Advil you have a choice to take the drug that best treats your symptoms. We need to support companies as they approach the finish line so that the decision on what treatment you or your child is prescribed is a decision you and your physicians make. Not a situation where you have no options.
Update of the Duchenne Care Considerations
PPMD’s Kathi Kinnett and Annie Kennedy have spent the last two days in Atlanta at the CDC working on an updated edition of the Care Considerations. Read Kathi’s blog to find out what the extraordinary team of Duchenne care experts assembled at the CDC have been focused on and what changes will be in the next iteration of the Care Considerations.
Don't Let Critical Senate Legislation Stall!
On Friday, September 25th, patient advocates from across the country will unite in a day of action to urge the Senate to make medical innovation a top priority. Please help us make a difference by calling your Senators and using social media to get their attention. A delay in this legislation is a delay in life-saving treatment for patients.
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 FAQs 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.
End Duchenne eNews: We See Strength in Teamwork
Catch up on the latest research and community updates in this month's End Duchenne eNews!