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Seizing the moment
December 2016
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2016. It is a year for the history books, especially for our community. And for many of us, a year we just want to put behind us.

The accelerated approval of EXONDYS 51 by the FDA is clearly one of the most significant events of the year. But it’s not the only thing! I talk often about the momentum in our community in research, care, and advocacy. On all fronts, the fight to end Duchenne continues to gain traction. And with the recent breakthroughs in CRISPR/Cas9 gene-editing technology, we have another exciting moment in our community—the opportunity to fund potentially game-changing research.

PPMD has a long history of funding innovative technology that hopes to stop the progression of Duchenne in all people living with the disease. CRISPR/Cas9 shows such promise—to target and modify DNA with incredible accuracy.

That is why PPMD is raising $250,000 to further the exploration of CRISPR/Cas9 technology in Duchenne. And that is why the Killian family is matching every gift, dollar for dollar, until we hit that goal. Donate today and your gift will be doubled!

Last week, PPMD marked #GivingTuesday by surpassing our goal of $20,000 to grow our ongoing efforts to support research into new clinical therapies. We ask for your help and you always come through!

Now is our opportunity to find out just how much CRISPR/Cas9 can do to treat Duchenne. Let’s go beyond the headlines of this promising therapy and see what it can do for our children. Let’s seize this moment for every person living with Duchenne and raise $250,000 by 12/31. Donate today and the Killian family will match your gift!

Thank you for supporting PPMD this holiday season. Thank you for helping us seize this moment.

Warmly,


Pat Furlong
PPMD Founder & President


Research
Upcoming Webinar: A Closer Look at the Potential of CRISPR/Cas9 in Duchenne
As you know, this holiday season PPMD is raising money to further explore the potential of CRISPR/Cas9 in Duchenne. Building on our introductory webinar on CRISPR/Cas9 that we held over the summer, join us Thursday, December 15 at 2:00 PM ET for our second webinar on this exciting technology. PPMD again welcomes Dr. Eric Olson from the Department of Molecular Biology at UT Southwestern Medical Center to review the basics of gene editing and talk about some of the next steps in the research and development pathway.
PPMD Is Seizing This Moment to Explore the Potential of CRISPR/Cas9 in Duchenne
CRISPR/Cas9 gene-editing technology shows incredible promise to target and modify DNA. But as with any new technology there are many questions that must be answered about CRISPR/Cas9 before we can understand just how much it can do to end Duchenne. How stable is this dystrophin protein produced by CRISPR/Cas9? What are the safety risks? Learn how PPMD is working with researchers to better understand the potential of CRISPR/Cas9 technology in Duchenne.
Webinar Recording: NS Pharma's Exon 53 Skipping Program
You can now view the recording of the webinar PPMD and NS Pharma (US subsidiary of Nippon Shinyaku, Co., Ltd) hosted last month to discuss NS Pharma's Exon 53 Skipping Program. Dr. Clemens and Lauren Morgenroth joined us to review data from the Phase I study conducted in Japan and present the study design for the trial that is opening in the US later this month. Watch the webinar.
PhaseOut DMD Adds Trial Sites
Summit’s clinicaltrials.gov record has been updated with additional locations and contact information for our PhaseOut DMD clinical trial by clicking here. More information about PhaseOut DMD can be found at utrophintrials.com.
Other Research News
Duchenne Research Update
PTC Therapeutics Receives France's 2016 Prix Galien for Translarna
Catabasis Quarterly Update
Solid Biosciences Granted U.S. & E.U. Orphan Drug Designations for Lead Gene Therapy Candidate for Duchenne
The Latest Update on Translarna from PTC Therapeutics
Advocacy
21st Century Cures Crossed Over the Finish Line!
To everyone in our PPMD community who called, reached out, emailed, and met with your elected officials – WE DID IT! We are thrilled that Congress has passed the 21st Century Cures (21CC) bill – which includes almost every provision that our Duchenne community championed and led throughout this process. President Obama has said he’ll sign the bill as soon as it reaches his desk – and then 21CC will be the law of the land and PPMD will begin engaging on implementation efforts. Learn more about this incredible victory for our community.
The MD-CARE Act in Action!

The PPMD team attended and presented on a variety of Duchenne care and research topics at the Muscular Dystrophy Coordinating Committee (MDCC) meeting to help coordinate activities related to Duchenne across the National Institutes and with other Federal health programs and activities. Gathered was leadership from every federal agency with influence and leverage over Duchenne.

We must continue to keep Duchenne on the front lines of agency priorities. Please consider joining us February 12-14 in Washington DC for the 2017 PPMD Advocacy Conference. Your presence in DC is a critical part of our Duchenne community’s strength and impact going forward!

Join us in Washington, DC this February
Please join us February 12-14, 2017 in Washington D.C. for what is going to be another critical year in Duchenne advocacy. 2016 marked a tremendous victory in our collective history, the first FDA approval of a Duchenne therapy – EXONDYS 51. It is important to remember that you, our advocacy community, paved the road to this approval. This victory marks an important first step in a brave new world for Duchenne, but much work remains for the entire community to ensure development, review, and access to promising therapies. Every year, over 150 Duchenne advocates come to Washington in February to urge Congress to continue to make Duchenne a priority. We MUST keep Duchenne on the front lines of agency priorities and maintain our growing presence in D.C. Don't be intimidated. Before you meet with your members of Congress, PPMD will provide you with extensive training and support, so you go into your meetings feeling empowered and prepared. Register today!
Other Advocacy News
Understanding Insurance Determinations
Duchenne Drug Review and the Patients' Input
Meet the Families of 21st Century Cures
Community
Community Corner
Calendar for a Cure
Donate $25.00 to help find treatments and a cure for Duchenne and receive a beautiful, autographed 2017 CALENDAR FOR A CURE, featuring photographs of wild Montana by Dave Stalling, a Duchenne father and amazing photographer. To learn more, click here. All proceeds will benefit PPMD.
Run For Our Sons
Chicago Marathon 2017
The new year hasn’t even begun but spots for our 2017 Bank of America Chicago Marathon team are almost sold out! Join us on Sunday, October 8, 2017 for the race’s 40th anniversary and go the distance to end Duchenne. Many of our past participants have called this their favorite marathon of all time – and with good reason! The fast, flat course through Chicago’s scenic and historic neighborhoods are lined with some of the best spectators around. Register today before our remaining entries are gone.
Rite Aid Cleveland Marathon
We’re heading to Cleveland once again for the 2017 Rite Aid Cleveland Marathon! Join us on May 20 and 21 for the 5K, 10K, Half Marathon & Full Marathon races, as well as a fun post-race team party!
We can’t wait to see you there.
Bank of America Shamrock Shuffle
Whether you’re new to running or a seasoned racer, the Bank of America Shamrock Shuffle is the perfect race to kick off your spring running season! This 8K race on April 2, 2017 through the streets of Chicago promises to be one of the most fun races you will ever do! Join our team.
Now recruiting:
Walt Disney World Marathon Weekend - January 5-8, 2017
Star Wars Half Marathon – The Light Side - January 12-15, 2017
Chevron Houston Marathon & Aramco Houston Half Marathon - January 15, 2017
Rock 'n' Roll New Orleans Marathon, Half Marathon & 10K - February 5, 2017
Rock 'n' Roll Raleigh Marathon, Half Marathon & 5K - April 1, 2017
Back of America Shamrock Shuffle 8K - April 2, 2017
Bank of America Chicago Marathon - October 8, 2017
Upcoming Events
• December 15, 2016: Happy Hour For A Cause
• January 5-8, 2017: Walt Disney World Marathon Weekend
• January 12-15, 2017: Star Wars Half Marathon – The Light Side
• January 15, 2017: Chevron Houston Marathon & Aramco Houston Half Marathon
• January 21, 2017: Every Single [One] Tour - Los Angeles
• February 5, 2017: Rock 'n' Roll New Orleans Marathon, Half Marathon & 10K
• February 18, 2017: UF PT Golf Tournament
Check out our full Calendar and sync it with your personal calendar!
Education
Join PPMD's Every Single [One] Tour in Los Angeles, CA!
Registration for the next stop on PPMD's Every Single One Tour is now open! Join us at Children’s Hospital Los Angeles on Saturday, January 21 for the latest updates in Duchenne research, care, and advocacy
How PPMD’s Certified Duchenne Care Centers Are Improving Quality & Access to Care
People with Duchenne are being cared for by clinics both near and far. Every one of them deserves the best care and treatment possible. Learn how PPMD’s Certified Duchenne Care Center program is making comprehensive Duchenne care more accessible and available for our families.
Duchenne Carrier Study at Nationwide Children’s Hospital
There is widespread consensus that female carriers of Duchenne are at risk for symptoms of muscular dystrophy. But what are those risks? PPMD is excited to support this important study at Nati onwide Children’s Hospital, which will evaluate the effects of a lower level of dystrophin in female carriers in three meaningful areascardiac, musculoskeletal, and psychosocial. We hope the results of this study will help us better understand the implications of being a Duchenne carrier, and possibly apply some of the answers to our understanding of Duchenne in males. Learn more about this study.
Webinar Recording: Deflazacort Access - Navigating Potential Changes to Your Prescription
Last month, PPMD hosted a webinar discussion with Masters Pharmaceuticals around the transition from Masters’ deflazacort to Marathon Pharmaceuticals deflazacort, should Marathon receive FDA approval in the U.S. on or around their anticipated February PDUFA date. Masters patients have a transition program available to them and PPMD has worked with both Masters and Marathon to put together a resource we hope will answer some of the questions that have arisen concerning the potential approval. Watch our webinar recording or read our blog to learn more.
New Paper Further Validates Need for Annual Cardiac MRI Surveillance in Duchenne
When PPMD convened the Contemporary Issues in Duchenne Cardiology meeting with NHLBI in July 2014, there were several recommendations. The strongest recommendations included the use of cardiac MRI as the gold standard for cardiac surveillance and starting ACE inhibitors with either the the first findings of cardiac fibrosis or dysfunction (or by age 10yo if findings remained normal). The two-year study described in a new paper titled "Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy" substantiates those recommendations. Because fibrosis occurs much sooner than cardiac dysfunction, and because it is not possible to see fibrosis by imaging the heart with echosonography, we hope that this paper will further validate the need for at least annual cardiac surveillance using MRI in patients living with Duchenne or Becker.
SAVE THE DATE! PPMD’s 2017 Calendar
In an effort to reach every single family facing a Duchenne diagnosis in the U.S., PPMD has launched a multi-year community experience called the Every Single [One] Tour. Focusing on each of the pillars that make up PPMD’s mission – To End Duchenne – the Every Single [One] Tour brings updates on research, advocacy, and care to cities across the country. Visit www.ParentProjectMD.org/Tour to view PPMD's 2017 Every Single [One] Tour schedule & find a stop near you!
More Education Updates
[FOR CLINICIANS] The Emergent & Acute Management of Patients Living with Duchenne
PPMD Certifies Center for Duchenne Muscular Dystrophy at UCLA
The Assistance Fund Launches Duchenne Program to Support Access to Exondys 51
The ‘JB's Keys to DMD’ Scholarship Fund at Northeastern University
Simple Actions
Seize this Moment
PPMD has a long history of funding innovative therapies that hope to stop the progression of Duchenne. Finally getting a Duchenne drug approved was an incredible victory. And with the recent breakthroughs in CRISPR/Cas9 gene-editing technology, we have another exciting moment in our community—the opportunity to fund potentially game-changing research.

That is why, for our year-end research project, PPMD is raising $250,000 to further the exploration of CRISPR/Cas9 technology in Duchenne—and that is why the Killian family is matching every gift, dollar for dollar, until we hit that goal.

Let’s #SeizeThisMoment for every person living with Duchenne! Help us find out just how much CRISPR/Cas9 can do to end Duchenne.
Our mission:
To end Duchenne. We accelerate research, raise our voices in Washington,
demand optimal care for all young men, and educate the global community.
CONTACT
Parent Project Muscular Dystrophy
401 Hackensack Avenue, 9th Floor
Hackensack, NJ 07601
t. 800-714-5437
SITES
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