Duchenne Action Month

FDA Announces Pediatric Advisory Committee Meeting

On May 18, FDA's Pediatric Advisory Committee will convene to consider the issue of allowing in-dwelling ports in Sarepta’s Essence trial. Details including instructions on submitting both written and oral testimony have now been posted by the FDA.
April 24, 2017 | Learn more 

Duchenne Action Month

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.
April 19, 2017 | Learn how you can get involved 

Upcoming Webinar

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.
April 18, 2017 | Learn more 

Certified Duchenne Care Center

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)!
April 6, 2017 | Learn more 

News Update

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.
April 5, 2017 | Learn more 

News Update

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.
April 5, 2017 | Learn more 

News Update

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.
March 29, 2017 | Learn more 

News Update

Action Alert: Make Your Voice Heard on New Healthcare Proposal

Urge Congress to protect the Duchenne community's healthcare priorities.
March 21, 2017 | Learn more 

News Update

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.
March 20, 2017 | Learn more 

News Update - eNews

End Duchenne eNews: Every Single [Connection]

Catch up on the latest research and community updates in this month's End Duchenne eNews.
March 17, 2017 | Learn more 

News Update

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.
March 16, 2017 | Learn more 

News Update

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!
March 14, 2017 | Learn more 

News Update

Webinar Recording: Idebenone Clinical Trials in Duchenne

PPMD and Santhera Pharmaceuticals recently hosted a webinar to discuss idebenone clinical trials in Duchenne.
March 13, 2017 | Learn more 

News Update

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.
February 27, 2017 | Learn more 

News Update

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.
February 24, 2017 | Learn more 

Certified Duchenne Care Center

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!
February 16, 2017 | Learn more 

 
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