view this email online
Parent Project Muscular Dystrophy Donate Now
Strength Happens Together
May 2014
Facebook Twitter RSS Feed Youtube

This is always one of the busiest and most exciting times of year for PPMD. We are just weeks away from what promises to be our most incredible, comprehensive Connect Conference to date. What an appropriate way to mark 20 years in the fight to end Duchenne!

If you've been to the Connect Conference before, you know that this is your opportunity to hear from the top minds in the Duchenne space. You have access to scientists, researchers, physicians, care providers, and industry – people who have dedicated their professional life to helping your child.

It's also about connecting with each other. I am always amazed at the amount of talent and the progress being made in Duchenne. But what excites me most about the conference, year after year, is seeing you and your family. Meeting families, watching kids interact and be kids, catching up with old friends – it's the highlight of my year and I hope to see you there!


Pat Furlong
PPMD Founder & President


Research
20th Annual Connect Conference – Agenda Now Available
For the latest research updates from the leaders behind all the exciting projects in the therapeutic pipeline, make sure you register for PPMD's 20th Annual Connect Conference in Chicago, June 26-29, 2014. Click here to download the agenda and register you and your family today. We look forward to seeing you in Chicago!
New Duchenne Therapies in Development
Treating Duchenne will take a "cocktail" approach and mutation-specific approaches like exon skipping are not our only irons in the fire. Many other therapeutic approaches will be entering the clinic by the end of this year or the next, providing a variety of options for participating in trials. Read more about what therapies are in the works in the latest blog from PPMD's Vice President of Research, Sharon Hesterlee, PhD.
DuchenneConnect Annual Report:
Registry Growth & Expanded Services
In 2013, the DuchenneConnect registry recruited for almost TWICE the number of clinical trials and research studies as 2012. Check out more DuchenneConnect highlights from this past year, and make sure you are registered with DuchenneConnect so you can be connected with actively recruiting research studies and clinical trials!

If you are already registered, please login to your account and update your/your child's medical history.
Results of PPMD-Funded Study Prompt Phase III Study of Tadalafil in Duchenne
Based on the results of a recent PPMD-funded study, Eli Lilly has begun a Phase III study to determine if tadalafil, a drug typically prescribed for erectile dysfunction or increased pressure in the arteries, can slow the progression of Duchenne. If tadalafil proves successful in slowing the progression of the disease it may be a good candidate to combine with other types of therapy that are mutation-specific or target other consequences of the loss of dystrophin in muscle cells. Learn more.
Sarepta Therapeutics Announces Plans to Submit NDA
Sarepta announced that its discussions with the Food and Drug Administration (FDA) over the last several months have borne fruit–the FDA has agreed to allow Sarepta to submit a New Drug Application (NDA) for eteplirsen. The company plans to submit the application by the end of this year and should have a decision from the FDA by mid-2015 about whether or not it will be approved. Find out what is next in this process and what this news may mean for your child and listen to Sarepta's Duchenne Development Program Update (webinar recording).
Other Research News
Researchers at the University of Michigan are interested in collecting patient impressions of how genetic neuromuscular disease affects their lives, particularly issues such as pain, depression, fatigue, sleep and cognitive problems.
Santhera Announces Successful Outcome of Phase III Study with Catena®/Raxone® in Duchenne
Halo Files "Complete Response" with FDA regarding Clinical Hold of HT-100 for Duchenne
Prosensa Update on Drisapersen: Re-dosing plans are underway and 96-week data from an open-label extension study of drisapersen for the treatment of Duchenne was presented at the American Academy of Neurology (AAN).
Advocacy
Unprecedented Collection of Duchenne Experts Developing Guidance for FDA
We have an unprecedented collaboration of over 80 expert stakeholders from all facets of the Duchenne community currently working on the Draft Guidance on Duchenne for the FDA! Check out a full list of participants in the latest update from our Director of Advocacy, Ryan Fischer. The delivery of this Draft Guidance to the FDA in the coming weeks will be a groundbreaking moment in our community. Later this month, the Draft Guidance will be posted online and made available for full public comment by you, the community at large. Stay tuned for your chance to voice your comments on the guidance.
MD-CARE Act Results:
Exon Skipping from Lab to Patient
Originally signed into law in 2001, the MD-CARE Act has birthed key discoveries in the muscular dystrophies, including the scientific justification for why exon skipping therapies could potentially work in humans. Without it being passed again in 2014, we risk a drastic reduction in potential scientific discoveries like we have seen over the past decade. Tell Congress to pass the MD-CARE Act this year and help us continue these advancements in research that are getting us closer to effective therapies and treatments for Duchenne. Learn more and take action now!
Other Advocacy News
In Support of Approvals: PPMD is doing everything it can to enable the FDA to speed the approval of new therapies to all who so desperately need them. Here are 3 simple actions you can take to help.
Community
One More Reason to Register for PPMD's 20th Connect Conference
Every year on the Saturday night of the Connect Conference, PPMD hosts a final dinner. Usually there is a speaker or two, dinner and drinks and a last moment to talk with friends. Not anymore! This year we are hosting a party – a celebration for all – to Celebrate Community. This party will be a great night complete with DJ, photo booth, face painters, tattoo artists, games, and activities. Learn more and register to attend!
Run For Our Sons
Be a Superhero!
We have entries left for Disney's Inaugural Avengers Super Heroes Half Marathon & 5K, and we need YOUR superpowers! Be part of something magical in Disneyland on November 15-16, 2014. Join our team today. Every runner will receive a special commemorative Run For Our Sons cape to mark our 10th year of running to end Duchenne.
Award-Winning Fundraiser
Congratulations to parent and PPMD Board Member Heinrich Meermann for being the 2014 Chevron Houston Marathon's 3rd Overall Top Fundraiser! Heinrich raised $26,800 and this was his 2nd year being a top 3 fundraiser for the Houston Marathon. Thank you for working so tirelessly for our community Heinrich! Registration for our 2015 Chevron Houston Marathon team opens on June 4th so check back to our race calendar then to sign up!
Inspiring Finish
Thanks to our teams who ran the Flying Pig Cincinnati Marathon and the DICK's Sporting Goods Pittsburgh Marathon earlier this month! Together they raised nearly $10,000 to help end Duchenne. Check out this video of Billy Ellsworth, son of runner Terry Ellsworth, as he runs across the finish line of the Pittsburgh race!
Now recruiting:
Rock 'n' Roll Seattle Marathon & Half Marathon - June 21, 2014
San Francisco Marathon - June 27, 2014
Disneyland Half Marathon Weekend - August 29 – 31, 2014
Bank of America Chicago Marathon - October 12, 2014
GoodLife Fitness Run Victoria Marathon - October 12, 2014
Baltimore Running Festival - October 18, 2014
TCS New York City Marathon - November 2, 2014
Avengers Super Heroes Half Marathon Weekend - November 14 – 15, 2014
Walt Disney World Marathon Weekend - January 8 – 11, 2015
Rock 'n' Roll New Orleans Marathon - January 25, 2015
And Save the Date for these popular 2015 races!
• Chevron Houston Marathon & Aramco Houston Half Marathon - January 18, 2015
Simple Actions Have Lasting Impact
One of the best resources for families at any stage of their Duchenne journey – and perhaps one of our greatest accomplishments as an organization – is PPMD's Annual Connect Conference. What started 20 years ago as a small group of parents gathering to discuss current Duchenne topics, has evolved into the largest, most comprehensive annual, international conference focused entirely on Duchenne.

Our meetings are an opportunity for families, physicians, researchers, and experts of all kind to speak face-to-face about Duchenne. These meetings open communication channels, lay the groundwork for future collaborations, and shape our understanding of the Duchenne landscape.

We hope you and your family will join us June 26-29 in Chicago for this life-changing experience. Today is your last day to register at our general rate. As of May 16, registration fees will go up. We look forward to connecting in Chicago next month!
Upcoming Events
• May 17, 2014: Team Aiden 5K
• May 17-18, 2014: Kaiser Permanente Colfax Marathon
• May 18, 2014: Rite-Aid Cleveland Marathon
• May 18, 2014: The Millennium 5K 4 Jake's Sake
• May 27-30, 2014: Spike Duchenne
• May 31, 2014: Kids Dash for Micah's Miracle
• June 21, 2014: Rock 'n' Roll Seattle Marathon & Half Marathon
• June 26-29, 2014: 2014 Annual Connect Conference
Check out our full Calendar and sync it with your personal calendar!
Connect Conference: Register Now!
2014 Connect Conference in Chicago
Date: June 26-29, 2014
Location: Chicago Marriott Downtown Magnificent Mile
Today is your last day to register at our general rate. Late registration fees go up May 16th!
Education
Certain Medicaid Waiver Payments May Be Excludable From Income
The Internal Revenue Service (IRS) recently reversed a policy and now allows parents of people with disabilities who receive Medicaid Home and Community Based waiver funds, for care giving services provided to their children, to exclude those funds from their incomes. These changes may apply to parents caring for a person with Duchenne, depending on available state programs and the eligibility of the parent. Learn more.
Living Duchenne: Ongoing Traumatic Stress
In her April blog, Living Duchenne, Colorado/Wyoming FACES Coordinator Ivy Scherbarth looked at the continuing stress of a Duchenne diagnosis in the life of a family, and the need to choose joy over trauma. If you didn't read this incredible blog last month, check it out and then tell us how you find the joy in a stressful and emotionally draining day.
EZ Lap Tray – A Father & Son's Labor of Love
Zack Heger has Duchenne. Zack and his parents struggled to find a comfortable way for Zack to sit up without limiting the use of his hands. They had to get creative with a variety of materials to come up with "Zack's tray." Out of all of the materials, high density foam was the best. It worked and EZ Lap Tray was born! EZ Lap Tray is a revolutionary new design for people of all ages, including those with physical disabilities or limited mobility. The tray provides comfort, stability, and independence. Visit ezlaptray.com to learn more.
A New Care Guide to Give to Your Provider –
Imperatives for Duchenne Care
Health care providers care for many people, with many diagnoses – far too many for any one provider to be an expert in them all. Make sure that you are receiving the best possible care by giving your provider this NEW "cheat sheet" guide to comprehensive Duchenne care.
More Education Updates
PPMD just launched our Certified Duchenne Care Center Program and is proud to award Nationwide Children's Hospital as our first Certified Duchenne Care Center. Find out more about the program's goals, strategy, and next steps.
Education, Behavior, and Duchenne: PPMD FACE Coordinator and Education & Behavior Specialist, Jill Castle, has launched a blog for families to provide behavioral and education support.
Simple Actions
PPMD's STIR program is a monthly giving opportunity, 100% of which goes towards funding research. This is a chance for not just you, but your entire family, to support potential therapies at a dollar amount you are comfortable with. In fact, we have a few PPMD families where every single relative (grandparents, aunts, uncles, cousins, and siblings) are giving. These are families that may not be able to pledge a large gift to PPMD. But by joining STIR and encouraging others to do the same, their combined monthly gifts are having an incredible impact.

Consider STIR and know exactly where your gift goes.
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
Facebook Twitter RSS Feed Youtube
© Parent Project Muscular Dystrophy 2014 — All Rights Reserved.

Help us end Duchenne. Donate today.
Forward to a friend. Was this e-mail forwarded to you? Subscribe today.

Unsubscribe from receiving email, or change your email preferences.


Unsubscribe from receiving email, or change your email preferences.