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.
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!
PPMD Recommends Marathon Convene Broad Array of Duchenne Stakeholders to Discuss Re-evaluation of Deflazacort Pricing
In light of the pause in commercialization of EMFLAZA™ (deflazacort) by Marathon Pharmaceuticals earlier this week, PPMD recommends that Marathon seek input from a broad array of stakeholders including Duchenne clinicians, health insurance plans, pharmacy benefit managers, and Duchenne advocacy groups.
Urge Congress to Protect Duchenne Community's Healthcare Priorities
This week, PPMD advocates from across the country are storming Capitol Hill to ensure legislators make Duchenne and rare disease a priority.
This year’s agenda includes urging Congress NOT to repeal the Patient Protection and Affordable Care Act until there is a guarantee that people with Duchenne will continue to have access to equal or better coverage. Please take a moment today to tell Congress why this matters so much to our community!
Marathon Pharmaceuticals Update on EMFLAZA™ (deflazacort)
Marathon has announced that they will pause the commercialization of their recently FDA-approved steroid EMFLAZA™ (deflazacort).
This pause will allow Marathon to consider the feedback they have received from the community since last week’s announcement regarding pricing and potential for combining this therapy with other approved drugs in the future. Marathon has assured families that during this pause, access to deflazacort will not be disrupted.
Deflazacort Approved in the U.S. for Duchenne
Today, the FDA approved EMFLAZA™ (deflazacort) for Duchenne. This is the first approval of a steroid for Duchenne and a therapy that applies to people with Duchenne 5 years of age and older, regardless of genetic mutation. Representatives from Marathon Pharmaceuticals will be joining us for a webinar Friday, February 10th at 12 PM EST to provide information about EMFLAZA’s FDA-approved label, patient support services, and access to this medication.
PPMD to Host Forum on Access to Emerging Therapies
PPMD is hosting the Access to Emerging Therapies Forum on February 13th, 2017 for our patient community and relevant stakeholders. Our presenters include policy experts who will present on the various points on the access continuum.
[Webinar Recording] MoveDMD Trial: Catabasis Provides Update
The Catabasis team recently joined us to discuss what they have learned from Part B of the MoveDMD® trial of edasalonexent (CAT-1004) in Duchenne and what comes next in the MoveDMD program.
[Webinar Recording] Mission DMD: FibroGen’s Anti-Fibrosis Program
FibroGen recently joined us for a webinar discussion on the company's investigational drug Pamrevlumab (FG-3019), along with FibroGen’s Mission DMD program that is currently being conducted to investigate Pamrevlumab in Duchenne.