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Spring Into Progress
April 2017
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I can't believe we are already halfway through April – 2017 seems to be flying by! I look at the content of this newsletter and it is overwhelming to see the ongoing momentum in research, advocacy, and care. I am reminded of early newsletters from years ago that featured one research project and maybe an Action Alert. Now we are seeing real progress being made on all fronts because of the passion and dedication of this community. When we work together, there is nothing we can't do.

Speaking of coming together...we are so excited about the number of families already registered for PPMD's Annual Connect Conference in Chicago this June. Many of you have taken advantage of our lower rates and packed agenda. If you haven't signed up yet, make sure you do as soon as possible so that you can be part of this incredible experience.

Later this week we will be rolling out an exciting project for the fall that we hope you will share with us. It is an opportunity to tell the world about Duchenne, raise awareness, and hopefully bring us one step closer to the day we end Duchenne.

I hope you enjoyed your Spring Break and all the wonderful holidays of the last couple of weeks. This time of year is full of promise and new beginnings. Take a minute for yourself to recharge your batteries, thaw out from the winter, and experience the hope of spring.

Warmly,


Pat Furlong
PPMD Founding President & CEO


Research
CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice
PPMD is excited to see promising news regarding the potential of CRISPR gene editing technology in Duchenne. These findings by Dr. Eric Olson and the Department of Molecular Biology at UT Southwestern Medical Center are the first to show the efficiency of Cpf1-mediated correction of genetic mutations in human cells and an animal disease model – providing us with a promising new tool in the CRISPR toolbox. Thanks to your support, PPMD was able to award a $250,000 grant to Dr. Olson and his lab earlier this year, as part of PPMD's gene transfer initiative. Read more about this exciting research.
PTC Therapeutics Announces Agreement to Acquire Emflaza™ (deflazacort)
PTC Therapeutics has entered into an asset purchase agreement with Marathon Pharmaceuticals, LLC to acquire all rights to Emflaza™ (deflazacort). While our concerns and questions remain the same and our recommendation for a path forward remains unchanged, PTC has a long history with our community, and we are optimistic that this news signifies that the company will ensure that deflazacort will be made available as a therapeutic option to our entire Duchenne population, including families that were unable to import it from overseas. We look forward to engaging with PTC as they move Emflaza forward. Read more about what this acquisition means and check out the latest community update provided by PTC.
Akashi Therapeutics Receives FDA Clearance to Resume HT-100 Clinical Development
PPMD is pleased to learn that the FDA has completed its review and concluded that Akashi may resume clinical development of HT-100 (delayed-release halofuginone) in people with Duchenne. Preventing fibrosis is an important target and an essential piece of a combination of therapies that will be required to end Duchenne. To learn more about HT-100 and next steps for the trial, read more or check out PPMD's recent webinar with Akashi.
ReveraGen BioPharma Receives FDA Fast Track Designation for Vamorolone
ReveraGen BioPharma Inc, announced that the FDA has granted Fast Track designation for vamorolone (VBP15) for the treatment of Duchenne. This designation can speed the review of efficacy and safety data for vamorolone in people with Duchenne, potentially leading to more rapid regulatory approval. PPMD is pleased to learn this news and happy to have provided early funding for this therapy. Read the release.
Summit to Extend Ongoing PhaseOut DMD Clinical Trial of Ezutromid in Patients with Duchenne
Summit Therapeutics announced that it will proceed with the planned extension phase of PhaseOut DMD, a Phase 2 clinical trial evaluating the utrophin modulator ezutromid, subject to regulatory approval. This follows an interim review of the safety and tolerability data from the ongoing trial by an independent Data Monitoring Committee and its support of Summit's plans to extend the clinical trial. The proposed extension phase will allow Summit to gather important long term safety and efficacy data of ezutromid in people with Duchenne that will likely be included in the data package for future applications as Summit continues to pursue regulatory approval of ezutromid. Learn more.
Bristol-Myers Squibb Enters into Agreement with Roche to License Anti-Myostatin Compound
Bristol-Myers Squibb (BMS) has entered into an agreement to license BMS-986089, an anti-myostatin adnectin in development for Duchenne, to Roche. To find out more, click here.
PPMD Hosts Duchenne Drug Development Roundtable Meeting Series, Part 3
PPMD recently convened the third meeting of our 3-part Duchenne Drug Development Roundtable (DDDR) meeting series: 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. Learn more.
Other Research News
Pat Furlong to Participate in 2017 Department of Defense's Congressionally Directed Medical Research Programs (CDMRP) as part of Duchenne Muscular Dystrophy Panel
Duchenne Research Update: February 2017
Solid Biosciences Raises up to $50 Million in Series C Financing
PPMD Webinar Archive
Advocacy
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. Once again, our community is at a pivotal moment in our history where we can affect change and improve the clinical trial experience for our children. We are grateful to the families and providers who have fought so hard to raise this issue up and to the FDA for understanding that this issue matters. 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. Details will be coming soon. Read more about this meeting.
Members of Congress Re-Introduce ABLE Improvement Bills
Great news – The ABLE program enhancements have been re-introduced in Congress and are gaining support. This package of bills include improvements to the original ABLE program that directly impact our community. Learn more.
At Long Last, PDUFA VI Hearings Have Begun – But There's a Twist
Wow! During one week in March, President Trump released his budget proposal, the American Health Care Act moves to the floor of the House for a vote today, the U.S. Supreme Court ruled on a landmark case around the rights of students with disabilities within the public school system, and at long last – after months of our Duchenne community's engagement, testimony, Hill visits, and discussions with Congressional Champions – the User Fee Hearings have begun. But like so much else these days, the User Fee negotiations took an unexpected twist. This 'plot twist' has made our community's engagement more important than we ever could have anticipated. Learn more.
PPMD Signs Appropriation Letter to Fund Newborn Screening
PPMD is proud to sign on to letters sent both to the Senate and the House asking that they provide at least $29.8 million to the Centers for Disease Control and Prevention's (CDC) Newborn Screening Quality Assurance Program (NSQAP) and at least $19.9 million to the Health Resources and Services Administration's (HRSA) Heritable Disorders program. This funding level will ensure states have the access to resources and technical expertise to support ongoing activities and implement new conditions to their state newborn screening panels. Read the letters.
PPMD Joins Letter to Protect Medicaid
In late March, PPMD joined 87 organizations to issue a letter calling on Congress to take a hard look at the likely significant and life-threatening consequences of the American Health Care Act on millions of patients. The organizations represent Americans with complex health needs who rely on Medicaid for access to care, prevention and treatment. While the AHCA is currently on hold, the same concerns remain when it is revisited. Click here to read the letter.
Other Advocacy News
Recap: PPMD's Adult Advisory Committee (PAAC) Leadership Meeting and 2017 Advocacy Conference
PPMD Joins Rare Disease Advocates in Letter to President
Meet the 2017 PAAC
Community
Run For Our Sons
Happiest Race on Earth
We're looking for more families to join us at the Happiest Place on Earth this Labor Day for the Disneyland Half Marathon Weekend! Say goodbye to summer and hello to one of the most fun race weekends of the year, featuring a 5K, 10K, half marathon, and Double Dare Challenge (10K and half marathon on consecutive days). There's something for everyone at this race through Disneyland and Disney's California Adventure theme parks. We hope to see you in California September 1-3!
Run Victoria
We've excited to be returning to British Columbia for the GoodLife Fitness Victoria Marathon on October 8! This race series around Victoria's scenic Inner Harbor offers a full marathon, half marathon, 8K Road Race, kid's race, and a marathon relay. Join us for a spectacular fall race weekend.
Shuffling On
The Run For Our Sons team Ran Fast and Lived Irish at the Bank of America Shamrock Shuffle 8K on April 2! Our 53 team members participated in the 8K race, as well as the new 2-Mile Walk through the streets and parks of downtown Chicago, and they showed their team spirit throughout the weekend. To date they've raised more than $62,000! Thank you to our runners and walkers for making this one of the year's most successful races. To see photos from the event visit our Facebook page.
Runner in the Spotlight
Thirteen-year-old Katie Napoli is one of our youngest "veteran" runners, having been with the team since she was 8 years old. Katie and her family run for Katie's cousin, Danny, and are part of one of our highest-fundraising teams in Run For Our Sons history. Katie's special relationship with her cousin has motivated her to run more than 21 races, from 5Ks to half marathons. She recently created an awareness video and blog featuring Danny and other young men living with Duchenne. Meet Katie, view her inspirational video, and read her blog, both of which are linked at the end of the profile.
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
TD Five Boro Bike Tour - May 7, 2017
Million Dollar Bike Ride - May 20, 2017
Rite Aid Cleveland Marathon - May 21, 2017
Rock 'n' Roll Seattle Marathon & Half Marathon - June 18, 2017
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
Upcoming Events
• April 22, 2017: 2017 Unionville Run For Our Sons 5K
• May 6, 2017: Luke's Ice House TX Fundraiser
• May 7, 2017: 2017 TD Five Boro Bike Tour
• May 7, 2017: Cincinnati Flying Pig Marathon
• May 20, 2017: Million Dollar Bike Ride
• May 21, 2017: Rite-Aid Cleveland Marathon, Half Marathon, 10K & 5K
• June 3, 2017: Every Single [One] Tour – Gainesville, FL
• June 9-10, 2017: Omaha Beer Fest
• June 10, 2017: #KOMD Captain Lawrence Charity Event
• June 18, 2017: Rock 'n' Roll Seattle Marathon & Half MarathonA
• 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
Check out our full Calendar and sync it with your personal calendar!
Education
Deflazacort Access Update from Masters
Masters has shared an update with the Duchenne community to provide information regarding its services to patients taking deflazacort in the US. Click here to read the letter.
Registration Open for PPMD's Annual Connect Conference
Registration is now open for PPMD's 2017 Connect Conference! This year's Connect Conference will be held in Chicago, IL from Thursday, June 29 to Sunday, July 2, 2017. We can't wait to see you and your family there! Learn all the exciting details of our biggest conference to date, including our lowest registration rate ever!
Complete the Bone Module
Collaborators from RTI have been busy analyzing data from DuchenneConnect around bone health. They plan to submit a paper soon for publication. We couldn't do this type of analysis without each of you keeping your information up-to-date. In April, we are asking all of our registrants to update their Bone Module. Log into your DuchenneConnect account, go to your profile page, and click on the Bone/Orthopedic Module. Either complete the survey for the first time, or select "Retake this survey" if you are updating your information. Please take 10 minutes to complete this survey and you could win a prize this month, including a new iPad!
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)! Led by Craig McDonald, MD and Associate Research Director, Erik Henricson, MPH, UC Davis has historically been a center widely acclaimed for their extraordinary neuromuscular research programs. We are thrilled to now recognize their provision of care and services, and include them in our growing network of comprehensive Duchenne care.
Every Single [One] Tour Recaps: Seattle & Iowa City
Earlier this month, PPMD hosted our second and third stops on our 2017 Every Single [One] Tour in Seattle, WA and Iowa City, IA. Thank you to all of our presenters, the extraordinary clinical providers at Seattle Children's Hospital and Iowa Children's Hospital, and our amazing families who joined us in these cities to hear the latest updates in Duchenne research, advocacy, and care. Read recaps from our meetings in Seattle, WA and Iowa City, IA. View PPMD's 2017 Every Single [One] Tour schedule to find a future stop near you!
Defining Patient Centricity
PPMD's Founding President & CEO, Pat Furlong, was part of a collaborative effort to define patient centricity and its important principles impacting the biopharmaceutical industry. The results of this study were recently published. Click here to read the published article.
More Education Updates
PPMD Designates Ann & Robert H. Lurie Children's Hospital of Chicago a Certified Duchenne Care Center
SAVE THE DATE! PPMD's 2017 Calendar
Simple Actions
Complete the Bone Module
In April, we are asking all of our registrants to update their Bone Module. Log into your DuchenneConnect account, go to your profile page, and click on the Bone/Orthopedic Module. Either complete the survey for the first time, or select "Retake this survey" if you are updating your information. Please take 10 minutes to complete this survey and you could win a prize this month, including a new iPad!
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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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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