September is Duchenne Action Month!
Mark your calendars…This year, PPMD is calling on the Duchenne community to save the month of September for Duchenne Action Month!
That’s right, this September will be packed full of simple and powerful actions you can take to help raise awareness and support PPMD’s work in the fight to end Duchenne.
Upcoming Webinar: Givinostat in Duchenne
Please join PPMD and Italfarmaco as we host a webinar Wednesday, April 26 at 1:00 PM ET to discuss Italfarmaco's investigational drug Givinostat, which is a histone deacetylase (HDAC) inhibitor.
PPMD Designates UC Davis a Certified Duchenne Care Center
PPMD is excited to announce our 15th Certified Duchenne Care Center -- UC Davis Department of Physical Medicine and Rehabilitation Neuromuscular Disease Clinic (UC Davis)!
PPMD Working with FDA to Plan Landmark Pediatric Advisory Committee Meeting to Improve Clinical Trial Experience
Later this spring, FDA’s Pediatric Advisory Committee will convene to consider amending the protocol in Sarepta’s Essence trial to allow the use of ports at the discretion of trial participants. If you have experience to lend we will be asking you in the coming weeks to submit your written testimony or join us in person at this Pediatric Ad Comm.
PPMD's Duchenne Drug Development Roundtable Meeting Series, Part 3: Clinical Trial Readiness from the Viewpoint of Clinicians and Infrastructure
Our hope is that this meeting, combined with the direction the first two meetings provided, will help us chart a new course and ensure that Duchenne clinical trials are optimized and available to every single one.
Webinar Recording: Akashi Provides Update on HT-100
PPMD and Akashi Therapeutics recently hosted a webinar to discuss HT-100 and next steps for the clinical program.
Action Alert: Make Your Voice Heard on New Healthcare Proposal
Urge Congress to protect the Duchenne community's healthcare priorities.
Action Alert: Urge Your Representative to Sign the FY 18 Duchenne Appropriations Letter
This action is critical to help ensure the agencies that impact Duchenne receive the funding they need to concentrate on the needs of our community.
End Duchenne eNews: Every Single [Connection]
Catch up on the latest research and community updates in this month's End Duchenne eNews.
PTC Therapeutics Announces Agreement to Acquire Emflaza™ (deflazacort)
Today we learned that PTC Therapeutics has entered into an asset purchase agreement with Marathon Pharmaceuticals, LLC to acquire all rights to Emflaza™ (deflazacort). Emflaza is the first treatment approved in the United States for all Duchenne patients five years and older, regardless of their genetic mutation.
Registration Now Open for PPMD's 2017 Connect Conference in Chicago, June 29 - July 2!
Join us for PPMD's 2017 Annual Connect Conference in Chicago, IL, June 29 - July 2, 2017. Register you and your family today!
Webinar Recording: Idebenone Clinical Trials in Duchenne
PPMD and Santhera Pharmaceuticals recently hosted a webinar to discuss idebenone clinical trials in Duchenne.
Webinar Recording: NS Pharma's Exon 53 Skipping Program
PPMD and NS Pharma recently hosted a webinar to provide an update on trial recruitment for NS Pharma's Exon 53 Skipping Program.
Duchenne Research Update: February 2017
Take a closer look at some of the incredible progress Duchenne research has made only two months into 2017. PPMD's SVP of Research Strategy, Abby Bronson, provides a snapshot of the Duchenne pipeline, including updates on therapies in development and PPMD's latest funding news.
PPMD Designates Lurie Children's a Certified Duchenne Care Center
PPMD is excited to announce our 14th Certified Duchenne Care Center -- Ann & Robert H. Lurie Children’s Hospital of Chicago!