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Let's Connect!
June 2017
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The Connect Conference is unlike any other experience in our community. It is the coming together of the greatest minds fighting Duchenne, with you – the families who navigate Duchenne 24/7 and have become experts yourself. Everyone who attends – either in person in Chicago this year or via our free live streaming or downloading presentations afterwards – does so because they are passionate about the fight to end Duchenne. We can all agree, we'd much rather exist in a world without this devastating disease. But until that day comes, this month's Connect Conference is our opportunity to unite, to lean on one another, to recharge our batteries.

The last twelve months have been extraordinary for our community. For every victory we have celebrated, we have been met almost immediately with a setback or backlash or both. But in these challenges, I think we have better defined who we are as a community, finding strength in our collective voice. This community is a force and this past year we have not only changed the landscape in Duchenne, but we have altered the entire rare disease terrain.

If you are attending this year's Connect Conference, we can't wait to see you! If you are unable to attend, don't forget to watch live streaming of most of the General Sessions starting June 29 through July 1. And, as always, we will upload all presentations by mid-July so that you can watch or re-watch or share some of the great information learned in Chicago.

In June, we also celebrate the amazing men in our community as Father's Day approaches this weekend. This year, let's not just honor dads, but also husbands, uncles, brothers, sons – the men that help you and your family fight against Duchenne every day. And what better way to celebrate these men than by making a donation to PPMD and taking advantage of our incredible board's extended matching gift this spring!

Many of you have heard me say over the years that none of us asked to be part of this community. If you're anything like me, you would have been perfectly happy never to hear the words Duchenne muscular dystrophy come from your doctor's mouth. But here we are. And because we are here together, we are stronger.

If I had to go on this journey with anyone, it is with YOU. And with YOU, we will end Duchenne.

See you in Chicago or online!

Warmly,


Pat Furlong
PPMD Founding President & CEO


Research
Stream PPMD's 2017 Connect Conference
Unable to attend this year's Connect Conference in Chicago? PPMD will be streaming a majority of the General Session presentations starting Thursday, June 29 through Saturday, July 1. Click here for details and a full agenda. If you can't stream the sessions or want to re-watch something, most presentations will be available for download by mid-July!
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, more research, and better care. Partnering with SOS will allow us to make a greater impact than we could alone. No matter where you are located, we encourage you to join DuchenneConnect today!
EMFLAZA FAQs & Informational Tour
PTC Therapeutics has provided an update regarding EMFLAZA. Click here to read the latest FAQs from PTC. The company will also be traveling around the country hosting informational meetings. For the current schedule, click here. Any additional questions or concerns, please visit EMFLAZA.com for answers to some of the most frequently asked questions PTC has received.
Other Research News
PPMD's 2017 Connect Conference Agenda is Now Available!
Akashi Therapeutics Receives FDA Clearance to Resume HT-100 Clinical Development
Read the Latest Community Update from Solid Biosciences
Pfizer Completes Enrollment of Phase 2 Study of Domagrozumab (PF- 06252616)in Duchenne Muscular Dystrophy
Santhera Provides Update on Timeline for Application of RaxoneŽ in Duchenne Muscular Dystrophy in Europe
Catabasis Quarterly Update
• Deciphering What We Know About EMFLAZA: (1) PTC Community Update; (2) PPMD BLog
PPMD Webinar Archive
Advocacy
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. The proposed bill would mean that states can decide who will receive Medicaid services and what services they will receive, and waitlists will grow exponentially. This is NOT acceptable for our community. Once Medicaid is dismantled, there will be no turning back. We need your help -- Take action now!
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. According to the determination, all conditions have been met and the protocol "may proceed with the inclusion of a totally implantable central venous access device (TICVAD)." We are grateful to our community. The families who fought so hard to raise this issue up and the kids who've endured despite great discomfort. Read the decision and learn about more about this historic meeting.
65 Members of Congress Send Letter of Strong Support for Duchenne Priorities for Next Year's Budget
The Duchenne advocacy community has once again helped deliver a strong FY 18 appropriations letter in both the House and Senate. The FY 18 House letter – led by Congresswoman Doris Matsui and Congressman Peter King – was signed by a total of 47 Representatives. The FY 18 Senate letter – led by Senator Roger Wicker and Senator Debbie Stabenow – was signed by 18 Senators. Click here for the list of signatures.
PTC Therapeutics Announces FDA Advisory Committee Meeting for Ataluren
PTC Therapeutics, Inc. announced that the FDA has notified the company of the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28, 2017 to review the new drug application (NDA) for ataluren (Translarna). This is another important moment for our community and we look forward to the opportunity to share our collective experiences with Translarna with the FDA. Click here to read more. As details on this Ad Comm become available, PPMD will let you know how you can get involved.
PPMD Signs On to DoD Funding Letters
Once again, PPMD has signed on to letters to leaders in both the House and Senate, requesting they continue to support the critical and highly successful defense health research programs funded through the Congressionally Directed Medical Research Programs (CDMRP) at the Department of Defense (DoD) for FY 2018. Click here to read the letters.
FDA Hiring Freeze Lifted
Recently, FDA Commissioner Scott Gottlieb announced that the hiring freeze, instituted on January 23, has been lifted. This reversal means that the FDA can begin to fill vacancies – needed resources for continued research into rare disease treatments and for the review and approval of new medicines. PPMD is proud to have been one of the 147 patient organizations, in an effort led by EveryLife Foundation, whose voice helped stress the importance of hiring sufficient and key personnel. Click here to read the letter sent to President Trump.
PPMD Illustrates Vision for Patient-Engaged Drug Development
PPMD's SVP of Legislation & Public Policy, Annie Kennedy, was recently interviewed by FasterCures for an article addressing Patient-Engaged Drug Development, and how it can help move the needle in the Duchenne community. Read the article here.
Other Advocacy News
Senate Introduces BENEFIT Act
PPMD's Pat Furlong Presents at Astellas Patient Advocacy Summit
PPMD Publishes & Testifies on Newborn Screening
PPMD Response to the House Passage of the American Health Care Act
Community
Duchenne Action Month
10th Annual Coach To Cure MD
on September 30th

Over the last 9 years, Coach To Cure MD has raised $1.5 million thanks to families like yours! Every dollar you have raised has helped us advance promising research, which led to two drug approvals for Duchenne in just the last year alone. We are less than four months away from the 10th Annual Coach To Cure MD on September 30, 2017 and YOU can help us break the $2 million mark this year to further our efforts and help us Tackle Duchenne! Join us from the moment college football kicks off through September 30, as thousands of coaches from hundreds of universities wear the Coach To Cure MD patch on their sleeve to raise awareness for Duchenne. Help celebrate 10 years of Coach To Cure MD and make this the year you get in the game and help us tackle Duchenne.
Run For Our Sons
Run Victoria
Now is the perfect time to register and start training for the GoodLife Fitness Victoria Marathon! Join us October 7 and 8 for this gorgeous race around Victoria's Inner Harbor. The race series includes full and half marathons, a marathon relay, an 8K, and a kids race, so it's perfect for seasoned runners and novices! Register and join us for an incredible weekend as we go the distance to end Duchenne.
Baltimore Running Festival
We're thrilled to once again be participating in one of our favorite East Coast races, the Baltimore Running Festival! This race series, which takes place October 21, includes both full and half marathons, a 4-person full marathon relay, and a 5K. The challenging courses will take you through Baltimore's historic neighborhoods, including the scenic Inner Harbor area and charming Fells Point. Join us on October 21 for this fun day of races!
Show Your Green
We had such a great time at this year's Bank of America Shamrock Shuffle, we couldn't wait to add it to our calendar for 2018! Registration is now open for this fun 8K race and 2-mile walk through downtown Chicago. Last year our team raised over $60,000 and had a fantastic time doing it! Join us on March 25, 2018 and help us kick off the spring race season!
Race to End Duchenne .1K
Have you signed on yet to put on a Race to End Duchenne .1K in your community? It's a super fun and easy action that we're asking people to take on this September for Duchenne Action Month. About the length of a football field, a .1K can be done almost anywhere and with any group of people! Plan one at your child's school, in your office, or in your community. We have How-To guides to help you along the way, and we're launching an online store in July that will have all the materials you need to plan the perfect race! Families across the country have signed on please consider joining them! Fill out our interest survey and download your How-To Guide to get started.
In Case you Missed It...
In an effort to maximize the amount of money going directly to our mission, PPMD has developed a new policy around applying funds raised through grassroots events to endurance event fundraising minimums. We believe this policy will help us stay on track while helping you put on the best fundraising events possible! Read the new policy here, and feel free to contact us with any questions.
Now recruiting:
Rock 'n' Roll Race Series - Nationwide, 2017 & 2018
Disneyland Half Marathon Weekend - September 1-3, 2017
GoodLife Fitness Victoria Marathon - October 8, 2017
Baltimore Running Festival - October 21, 2017
Rock 'n' Roll Savannah Marathon - November 4-5, 2017
TCS New York City Marathon - November 5, 2017
Walt Disney World Marathon Weekend - January 3-7, 2018
Star Wars Half Marathon - The Light Side - January 12-14, 2018
Chevron Houston Marathon & Aramco Houston Half Marathon - January 14, 2018
Bank of America Shamrock Shuffle - March 25, 2018
Upcoming Events
• June 16, 2017: Team Henry Fighting Duchenne
• June 17, 2017: Jared Sweet Golf Outing
• June 18, 2017: Rock 'n' Roll Seattle Marathon & Half Marathon
• June 19, 2017: Jumping Jack Golf Tournament
• June 24, 2017: UFL PTA Golf Outing
• June 29 - July 2, 2017: PPMD's 2017 Annual Connect Conference
• July 15, 2017: Volley for Mitchell
• July 29, 2017: Friar Fight to End Duchenne Golf Tournament
• August 19, 2017: Mitchell's Run thru Rockford
Check out our full Calendar and sync it with your personal calendar!
Education
PPMD's Every Single One Tour Recap: Gainesville, FL
Families from across Florida gathered together in Gainesville earlier this month to hear the latest updates in Duchenne research, care, and clinical trials. Thank you to all of our presenters and amazing families who joined us for an information-packed day. A full wrap-up (including links to presentations!) is now available on our blog. Next up, the 2017 PPMD Connect Conference later this month and then we're off to Charleston, WV on September 9!
PAAC Member Ben Dupree Discusses CRISPR on NBC's Sunday Night with Megyn Kelly
We are immensely proud of PPMD Adult Advisory Committee (PAAC) Member Ben Dupree and his mother, PPMD FACES Coordinator Debbie Dupree for their recent national appearance on Sunday Night with Megyn Kelly on NBC, talking about the hope with CRISPR (gene editing) technology. Earlier this year PPMD funded Dr. Eric Olson's lab to look at answering important questions in the early stages of research for this new technology. We are grateful to the community for supporting this research and look forward to hearing an update at the 2017 PPMD Connect Conference. Thank you, Ben and Debbie for your incredible advocacy!
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.
[WEBINAR] Nationwide Children's Hospital Carrier Study
Dr. Kan Hor and Dr. May Ling Mah joined PPMD for a webinar to provide an overview of the Nationwide Children's Hospital Carrier Study. Our speakers discussed cardiac disease in females who are carriers of the Duchenne genetic mutation – what we know, what we do not know, and what we hope to learn. If you missed it, you can now watch the recording of the webinar.
More Education Updates
PPMD Convenes Meeting to Explore the Burden of Pediatric Clinical Trials
SAVE THE DATE! PPMD's 2017 Calendar
Simple Actions
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!
Learn how you can get involved to help raise awareness and support PPMD's work in the fight 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
Parent Project Muscular Dystrophy
PPMD Community
DuchenneConnect
Run For Our Sons
Coach To Cure MD
CONNECT
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© Parent Project Muscular Dystrophy 2017 – All Rights Reserved.

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