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Every Single [Connection]
March 2017
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I know it's hard to think about summer in Chicago for the 2017 PPMD Connect Conference with a major chunk of the U.S. still buried in snow. But while March is known for crazy weather like we are experiencing now, it is also when we open registration for arguably the most impactful weekend our community shares together.

The 2017 PPMD Connect Conference marks the 23rd time families, physicians, researchers, caregivers, industry partners and those living with Duchenne have gathered to discuss all topics Duchenne. This conference is like no other. Here, the key is community. When this community comes together to learn, to listen, and to look forward at driving change, we become empowered and a force to be reckoned with. Now is the time for us to come together as a community and seize this moment.

So many of my closest friendships started at the Connect Conference, and as much as I look forward to the incredible presentations on research, care, and advocacy, it is reuniting with old friends that makes the Connect experience special.

Because you are our priority, and bringing us together is essential, PPMD is pleased to offer reduced registration fees for our 2017 Connect Conference. Thanks to the generosity of our donors and sponsors, we have been able to reduce the registration fee by approximately 50% from last year!

Please join us this June for the 2017 PPMD Connect Conference in Chicago, for what promises to be a life-changing experience for the entire family!

Warmly,


Pat Furlong
PPMD Founding President & CEO


Research
PTC Therapeutics Announces Agreement to Acquire Emflaza™ (deflazacort)
Yesterday we learned that 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 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. Learn more.
Duchenne Research Update: February 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. Take a closer look at some of the incredible progress Duchenne research has made only two months into 2017.
PPMD's Duchenne Drug Development Roundtable Meeting Series, Part 2: Optimizing Clinical Trials
According to ClinicalTrials.gov, there are over 50 interventional trials open for Duchenne. And while we couldn't be happier that there are so many "shots on goal," as a community we are committed to minimizing the length of these trials, the number of patients required, and the exposure to placebo – while maximizing the robustness of the results. In short, we want those shots on goal to yield "wins". From this meeting, we hope to have more direction as to the best path to pursue for optimizing clinical trials in Duchenne. Read a synopsis of the meeting.
[Webinar Recording] Idebenone Clinical Trials in Duchenne
Santhera Pharmaceuticals recently joined us for a webinar to discuss idebenone clinical trials in Duchenne. Dr. Thomas Meier provided an overview of the history of the idebenone development program, discussed the need to address respiratory decline in Duchenne, and explained the currently recruiting SIDEROS clinical trial of idebenone in boys using steroids. You can now watch the recording of the webinar.
[Webinar Recording] NS Pharma's Exon 53 Skipping Program
Last month, PPMD hosted a webinar to provide an update on trial recruitment for NS Pharma's Exon 53 Skipping Program. Dr. Clemens and Lauren Morgenroth reviewed the study design and provided the most recent updates for the trial that is ongoing in the US. Learn more.
Other Research News
PTC Therapeutics Announces FDA Acknowledgment of NDA Filing for Translarna for the Treatment of Nonsense Mutation Duchenne
Swiss specialty pharma company Santhera Pharmaceuticals announces opening of U.S. office in Burlington, MA
PPMD Awards $600,000 Grant to NJIT & Talem for Next Phase of Upper Extremity Exoskeleton
PPMD Awards $250,000 Grant to UT Southwestern Medical Center to Explore CRISPR/Cas9 Technology
PPMD's Duchenne Drug Development Roundtable: Bringing Industry Together to Accelerate Research
Deflazacort Approved in the U.S. for Duchenne
PPMD Webinar Archive
Advocacy
Recap: PPMD's Adult Advisory Committee (PAAC) Leadership Meeting and 2017 Advocacy Conference
As a member of the PPMD Adult Advisory Council (PAAC), Buddy Cassidy has testified at congressional briefings, helped to shape policy, and served as a mentor to others within our community. Most recently, Buddy and the rest of the PAAC attended PPMD's 2017 Advocacy Conference and PAAC Leadership Meeting in Washington, DC. Read Buddy's blog to learn more about his empowering experience in Washington with the PAAC and what this impressive group is working on next.
PPMD Joins Rare Disease Advocates in Letter to President
PPMD joined 200 patient organizations in a letter to President Trump about the challenges facing rare disease patients in America and the vital role that federal agencies play in helping to accelerate the research, development, review, and approval of treatments. Participating groups want the White House to understand that these agencies must have the ability to hire and retain personnel in order to accomplish their respective missions and to achieve the broader goal of treating and curing diseases. Click here to read the letter.
Other Advocacy News
Drug Developers Embrace the Rising Prominence - and Influence - of Patient Advocacy
ACTION ALERT: Urge Congress to Protect Duchenne Community's Healthcare Priorities
PPMD Hosts Forum on Access to Emerging Therapies
PPMD Participates in Newborn Screening Committee Meeting
Meet the 2017 PAAC
Community
Run For Our Sons
TD 5 Boro Bike Tour
We have a few entries left for the 40th anniversary TD 5 Boro Bike Tour on May 7th! Bike 40 miles through the streets of New York City's five boroughs completely traffic free! Not up for 40 miles? Shortcuts are available at miles 14 and 27. This race sold out within hours, but we're your ticket in. Visit our website to register, and bike the distance to end Duchenne!
Last Call for the Bank of America Shamrock Shuffle
There's still time to register and fundraise for your entry into the Bank of America Shamrock Shuffle 8K on April 2nd in Chicago! Our team is 45 members strong and we're looking to add a few more runners and walkers. This festive 8K race (that's about 5 miles) takes you on a fast and flat course through the streets of Chicago. There's also a 2 Mile Walk, so everyone can participate! Join our team now.
Ohio is the Place to be in May!
We're' excited to be participating once again in both the Cincinnati Flying Pigs Marathon on May 7th and the Rite Aid Cleveland Marathon on May 21st. Both of these races have multiple distances available, and we invite our Midwest families to join us for either one! We hope to see you in Cincinnati or Cleveland in May!
Runner in the Spotlight
Alpa Khushalani signed up for her first race, the Bank of America Shamrock Shuffle 8K, after hearing another Duchenne mom share how joining the Run For Our Sons team had helped her cope with her son's diagnosis. Having never run a race before, Alpa was nervous, but propelled by the support of friends and family, she is determined to do something to raise awareness and funds to end Duchenne. Alpa's team, Kudos for Krishna, has raised nearly $30,000 so far, and they are already planning their next race! Meet Alpa.
Now recruiting:
Back of America Shamrock Shuffle 8K - April 2, 2017
Cincinnati Flying Pig Marathon - May 7, 2017
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
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
• March 19, 2017: Shamrock Shuffle 5K Run-Walk
• March 26, 2017: Every Single [One] Tour – Seattle, WA
• April 1, 2017: Every Single [One] Tour – Iowa City, IA
• April 1, 2017: 2017 Rock 'n' Roll Raleigh Marathon
• April 2, 2017: Bank of America Shamrock Shuffle 8K
• April 8, 2017: Garrett's Hero Run at ETSU
• April 22, 2017: 2017 Unionville Run For Our Sons 5K
• 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
Check out our full Calendar and sync it with your personal calendar!
Education
Register Today for PPMD's 2017 Connect Conference in Chicago, June 29 - July 2!
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.
Join PPMD's Every Single [One] Tour in
Seattle, WA or Iowa City, IA!
It's not too late to join PPMD's Every Single [One] Tour events in Seattle on March 26th and Iowa City on April 1st! 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. We hope you can join us in Seattle or Iowa City!
Complete/Update PPMD's DuchenneConnect Breathing/Respiratory Module
Want to contribute to research from your home? You can make a difference by completing the Breathing/Respiratory survey on DuchenneConnect.org. Even if you have completed this last year, we ask that you log back into your account and retake the survey. Everyone who completes or updates this Module will be entered into our monthly raffle to win one of three prizes, including a new iPad!
PPMD Designates Ann & Robert H. Lurie Children's Hospital of Chicago 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! Led by Medical Director Dr. Nancy Kuntz, Lurie Children's boasts the Mazza Foundation Neuromuscular Program which offers specialized care to families and children with medically-challenging neuromuscular conditions. We are extremely happy to include Lurie Children's in our growing network of comprehensive Duchenne care. Learn more about this certified center.
Decode Duchenne Receives 500th Patient Application
Identifying people with Duchenne is critical in our fight to end this disorder so that optimal care is provided as soon after diagnosis as possible, as well as connecting patients to the most relevant clinical trials or approved therapies. PPMD could not be more appreciative or proud to collaborate with exceptional industry partners who continue to prove their commitment to the Duchenne community by supporting important programs like Decode Duchenne. Learn more.
More Education Updates
PPMD Designates Yale New Haven Children's Hospital a Certified Duchenne Care Center
Every Single One Tour – L.A. Recap
Simple Actions
Decode Duchenne
With the Duchenne community receiving our first FDA approvals ever in the last few months, the free testing, interpretation and counseling that is provided through PPMD's Decode Duchenne program is more important than ever before. Take advantage of this free program today.
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
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© Parent Project Muscular Dystrophy 2017 – All Rights Reserved.

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