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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 24, 2017 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 19, 2017 Category: Neurology Source Type: news
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)! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 6, 2017 Category: Neurology Source Type: news
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 per son at this Pediatric Ad Comm. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 5, 2017 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 5, 2017 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 29, 2017 Category: Neurology Source Type: news
Upcoming Webinar: Akashi Provides Update on HT-100
Join PPMD and Akashi Therapeutics as we host a webinar Monday, March 27 at 1:30 PM ET to discuss HT-100 and next steps for the clinical program. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 28, 2017 Category: Neurology Source Type: news
Action Alert: Make Your Voice Heard on New Healthcare Proposal
Urge Congress to protect the Duchenne community's healthcare priorities. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 28, 2017 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 20, 2017 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 16, 2017 Category: Neurology Source Type: news
Registration Now Open for PPMD's 2017 Connect Conference in Chicago, June 29 - July 2!
(Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 14, 2017 Category: Neurology Source Type: news
Webinar Recording: Idebenone Clinical Trials in Duchenne
PPMD and Santhera Pharmaceuticals recently hosted a webinar to discuss idebenone clinical trials in Duchenne. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 13, 2017 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - February 27, 2017 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - February 24, 2017 Category: Neurology Source Type: 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! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - February 16, 2017 Category: Neurology Source Type: news
