News Update

Webinar Recording - Gene Therapy for Duchenne

PPMD recently hosted a webinar focused on understanding the different therapies that are being developed that are commonly referred to as gene therapy, including micro-dystrophin and CRISPR/Cas9, how are they similar and different, and what are the challenges and limitations for each of them. If you missed it, the recording of the webinar is now available!
August 15, 2017 | Learn more 

Upcoming Webinar - August 9

Join PPMD's Every Single One Tour in Charleston, WV

It's not too late to join PPMD's Every Single [One] Tour in Charleston, WV on September 9! Combining each of the pillars that make up PPMD's mission -- To End Duchenne -- the Every Single [One] Tour brings free day-long meetings featuring updates on research, advocacy, and care to Duchenne families, professionals, and support networks across the country.
August 10, 2017 | Learn more 

News Update

Newly Published Data Validates Micro-Dystrophin Gene Therapy Approach in Animal Model for Duchenne

At the start of the year, PPMD made its biggest investment ever -- $2.2M to Jerry Mendell at Nationwide Children’s Hospital -- for a Phase 1 Safety trial of gene therapy in Duchenne. This new publication of data showing that the technology works very well in dogs gives us added evidence that gene therapy is ready to go into humans and supports our decision to invest heavily in this approach. While dogs and humans are not exactly the same, these results are so very encouraging. We look forward to working with Dr. Mendell and his team at Nationwide and Sarepta, our co-investors, to move this trial forward.
July 31, 2017 | Learn more 

News Update

PJ Nicholoff Steroid Protocol Published

We pleased to now announce that the PJ Nicholoff Steroid Protocol has now been published in PLOS Currents Muscular Dystrophy. It is our hope that this publication will make it into the reference library of every Duchenne and emergency room provider.
July 18, 2017 | Learn more 

News Update

2017 Connect Conference Resources & Recordings

Check out our online archive of presentations to review some of the incredibly informative panels and discussions from this year's meeting. And mark your calendars -- the 2018 Connect Conference will be held in Scottsdale, Arizona, June 28 - July 1!
July 17, 2017 | Learn more 

News Update

Save $10 on your Duchenne Action Month gear!

Restock your family's supply of PPMD merchandise so that you can raise awareness this September as part of Duchenne Action Month! Shop now and save $10 on your order of $60 or more.
July 12, 2017 | Learn more 

News Update

PPMD to Host Duchenne Drug Development Roundtable During 2017 Connect Conference

PPMD will host our annual Duchenne Drug Development Roundtable (DDDR) during the 2017 PPMD Connect Conference. We appreciate each company that has come to the table to lean in even further and we look forward to the opportunity to work together as a community to explore the innovations and opportunities that are raised through ongoing discussion and exploration.
June 30, 2017 | Learn more 

News Update

PPMD Convenes International Experts for Inflammation and Immunity in Duchenne Muscular Dystrophy Workshop

The agenda for this meeting includes Duchenne experts, as well as leaders from other disease areas bringing their expertise and experience to the Duchenne community. We look forward to better understanding the role that inflammation and immunity play in Duchenne.
June 28, 2017 | Learn more 

News Update

Join the 2017 #PPMDConnect Conference Online!

Unable to attend this year's Annual Connect Conference in Chicago? Join us online! PPMD will be live streaming most general sessions Thursday, June 29 - Saturday, July 1.
June 27, 2017 | Learn more 

News Update

Action Alert! Stop the Senate from Dismantling Medicaid

The US Senate version of the American Healthcare Act (ACHA) is currently being created behind closed doors. Medicaid is in serious jeopardy. This is NOT acceptable for our community. We need you to take action now!
June 17, 2017 | Learn more 

News Update

PPMD’s Every Single One Tour Recap: Gainesville, FL

Thank you to all of our presenters and amazing families who joined us for an information-packed day in Gainesville! A full wrap-up (including links to presentations!) is now available on our blog.
June 14, 2017 | Learn more 

news Update

DuchenneConnect Expands Down Under!

In collaboration with Save Our Sons duchenne foundation (SOS), we are happy to announce that DuchenneConnect Australia has launched! Having clinical trials in as many countries as possible helps the whole Duchenne community. We are stronger working together, pushing for more trials and research.
June 5, 2017 | Learn more 

News Update

Join the Coach To Cure MD team on September 30th!

Help us celebrate 10 years of Coach To Cure MD on September 30th! What started as a one-day event has become a season of raising awareness and the perfect way to finish Duchenne Action Month strong. Make this the year you get in the game and help us tackle Duchenne!
June 1, 2017 | Learn more 

News Update

FDA Makes Determination Regarding In-Dwelling Ports

We are pleased to announce that the FDA has made a positive determination on the referral of the ESSENCE protocol for review under 21 CFR 50.54.
May 26, 2017 | Learn more 

News Update

Finding Your Light: Fighting Depression in Duchenne

Children and teens with Duchenne, as well as their families, draw upon tremendous strength to cope with this life limiting diagnosis. It is normal to occasionally feel that it is too much to manage. It’s also important to recognize when worries or feelings of sadness are having a real impact on one’s ability to live life to the fullest. PPMD’s SVP of Clinical Care, Kathi Kinnett, has enlisted help from experts to put together a blog to help families identify signs of depression and recognize risk factors and warning signs of suicidal thoughts or intentions.
May 25, 2017 | Learn more 

News Update

Before Summer Kicks Off, Get Your Event Kicked Off!

Now is the time to start planning a Race to End Duchenne .1K at your school for Duchenne Action Month!
May 24, 2017 | Learn more 

News Update

Webinar Recording: Nationwide Children’s Hospital Carrier Study

PPMD recently hosted a webinar for a discussion on the Nationwide Children’s Hospital Carrier Study.
May 23, 2017 | Learn more 

News Update

FDA Pediatric Advisory Committee Unanimously Votes YES — Now Moves to Commissioner

Today’s Pediatric Advisory Committee Meeting resulted in a unanimous decision from committee members to recommend the use of in-dwelling ports in Sarepta's ESSENCE clinical trial. A final decision will be announced once the Commissioner has reviewed the summary report.
May 18, 2017 | Learn more 

News Update

Deciphering What We Know About EMFLAZA

The PPMD team is continuing to work with families to help them navigate access to newly approved therapies. Our latest blog attempts to clarify some of the questions that have arisen with the recent community update on EMFLAZA’s launch -- including those surrounding the “weight-based” pricing model and prior-authorization.
May 15, 2017 | Learn more 

News Update - Action Alert

ACTION ALERT: Urge Your Senators to Sign the FY18 Duchenne Funding Letter

Please urge your Senate members to sign the FY18 Duchenne funding letter. Help ensure the agencies that impact Duchenne receive the funding they need to concentrate on the needs of our community!
May 15, 2017 | Take action 

News Update

In Honor of Moms

This Mother’s Day, thank someone for their role in the fight to end Duchenne.
May 10, 2017 | Learn more 


News Update

Webinar Recording: Results from Phase I/II HOPE Clinical Trial of CAP-1002

PPMD, Coalition Duchenne, and Capricor recently hosted a webinar to hear a community update on the six-month results from Capricor's randomized Phase I/II HOPE Clinical Trial of CAP-1002.
May 9, 2017 | Learn more 

PPMD's Response to PTC Announcement of EMFLAZA (deflazacort) Pricing & Access

Today, PTC Therapeutics provided a community update on EMFLAZA (deflazacort) which they acquired earlier in the year from Marathon Pharmaceuticals. PPMD will continue to monitor and remain engaged in the access environment in order to understand the impact of every new approval, including EMFLAZA, and will update our online Access Resource as information becomes available.
May 8, 2017 | Learn more 

New Update

Meet the Race to End Duchenne .1K!

September is Duchenne Action Month, and now is the time for you to start planning an event than can raise awareness and money, while providing people with an amazing experience that is fun and rewarding…the Race to End Duchenne .1K!
May 5, 2017 | Learn more 

New Update

Webinar Recording: Givinostat in Duchenne

PPMD and Italfarmaco recently hosted a webinar to discuss Italfarmaco's investigational drug Givinostat and how it works in Duchenne.
May 1, 2017 | Learn more 

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 

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

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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