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September 2014
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Autumn. It'll be here in mere days. And we know that you and your family are in full back-to-school mode. In all of the craziness and stress of everyday life, we know that you can be overwhelmed with bits of news that you hear or read, questions about flu season preparation, the latest research and trials, the best care for your child. PPMD wants to remind you...we're here for you. Amidst the noise of our digital/social media/mobile age, take a breath and turn to PPMD with your questions and concerns. We won't always know the answer, but we will always do our best to get you an answer.

I couldn't be more proud of the team of talented individuals we have assembled at PPMD. And you should know that each and every person in this organization is here to help you and your family navigate Duchenne.

Try to enjoy the beauty of the fall, the kickoff of Coach To Cure season, and remember that your PPMD family is just a phone call or email away!

Warmest regards,


Pat Furlong
PPMD Founder & President


Research

PTC Completes Enrollment of Translarna™, Phase 3 Confirmatory Trial

PTC Therapeutics, Inc. announced last week that it has completed enrollment of ACT DMD, the Phase 3 confirmatory trial of Translarna™ (ataluren) for patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Top-line data from the trial is expected in the second half of 2015 and will support further approvals globally, following European approval received earlier this year. Read the press release.

UCLA's DNA Detectives in Action

This a great article on the work Dr. Stanley Nelson and Dr. Carrie Miceli are doing. We are so grateful to have Dr. Nelson and Dr. Miceli in our corner, fighting to end Duchenne. PPMD is currently funding Dr. Nelson's study to look for modifying genes that make the symptoms of Duchenne more or less severe. Read the article.

Promising New Gene Editing Method

Although much progress has been with techniques that tweak the way cells interpret mutations in the RNA message for dystrophin, like exon-skipping and stop codon read-through, now investigators at UT Southwestern Medical Center have made strides in permanently correcting mutations in the dystrophin gene itself. Learn more.
Other Research News
Prosensa announces Lancet Neurology publication of an exploratory phase II study (DEMAND II) demonstrating efficacy and safety of drisapersen in patients with Duchenne muscular dystrophy
Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key Therapeutic Efficacy Marker for Muscular Dystrophy
If you were unable to attend the 20th Annual Connect Conference in Chicago, June 26-29, 2014 or if there is a session you would like to revisit, you can now download most presentations by clicking here.
PPMD has a decade-long history of supporting exon-skipping, but what exactly is it? Our VP of Research, Sharon Hesterlee, PhD, explains.
PPMD Welcomes Two New Scientific Advisory Committee Members
Advocacy

Annie Kennedy Joins PPMD

We are thrilled to welcome Annie Kennedy, our new Senior Vice President of Legislation and Public Policy! Annie joins PPMD after being with the Muscular Dystrophy Association (MDA) for 25 years, first as a camp volunteer and as an employee for the last 16 years. Annie's focus will be on advancing public policy, addressing transition issues for people with Duchenne, and working on newborn screening issues. Read a blog from Annie about this new chapter in her life as well as a welcome message from our Vice President of Advocacy and Community Outreach, Ryan Fischer.
ACTION ALERT: Tell the Senate to Take Action on the MD-CARE Act!
Congress has returned from their Summer recess and it is time to re-engage them on the MD-CARE Act! We need each member of our community to reach out to your Senators and urge them to tell leadership to bring the MD-CARE Act to the floor for a vote. Learn how you can quickly and effectively help us get this critical legislation passed for all people with Duchenne.

FDA Guidance: Next Steps

The next stage in the Draft Guidance process has now officially begun! FDA had a closed door meeting on August 13 to discuss the Guidance submitted by PPMD in late June on behalf of the Duchenne community. On September 4, they officially announced that the Draft Guidance has been added to a docket and a 30-day public comment period has started. This is an important next step in the process of FDA creating their own draft guidance and this project continues to be a shining example that strength happens together! View the guidance.

PPMD Submits Cardiac Recommendations to FDA

PPMD and the National Heart, Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH) collaborated in convening a working group to explore contemporary issues in Duchenne cardiology (read a recap here). Attendees at that meeting included experts in pediatric and adult cardiology, clinical research, patient advocacy, and representatives of the FDA. During discussions at that meeting, the FDA indicated interest in receiving recommendations for cardiac surveillance and cardiac markers, to be included in current and future Duchenne clinical trials. In response to those questions, recommendations were developed by the Expert Working Group and submitted to the FDA for consideration for inclusion in the draft guidance. Almost immediately, we received a thank you for the recommendations and submitted them to the docket. Read more about what PPMD submitted.

PPMD's Ongoing Participation in 21st Century Cures

PPMD and the Duchenne community continue to have a seat at the table for 21st Century Cures Initiative. This effort by Congress is aimed at accelerating the pace of cures in America. Congress is looking at the full arc of this process – from the discovery of clues in basic science, to streamlining the drug and device development process, to unleashing the power of digital medicine and social media at the treatment delivery phase. Pat Furlong testified to the Energy and Commerce Committee in early July and now Congress is holding regional roundtables around the country to engage patients, professionals, and industry. We are proud to have Ellen Wagner, PPMD board member representing PPMD and the Duchenne community in IL with Congressmen Davis and Shimkus. Learn more.
Community
It's Time to Tackle Duchenne!
September 27 is the 7th Annual Coach To Cure MD. With coaching staffs for all major televised college football games wearing our CTC patches, this is the Duchenne community's chance to reach millions of spectators over a single weekend. Join the CTC team and learn how you can help us tackle Duchenne at CoachToCureMD.org.
World Duchenne Awareness Day
September 7, 2014 will be remembered in our community's history as the first-ever World Duchenne Awareness Day. This was a global effort and seeing the response around the world was awe inspiring. Visit the World Duchenne Awareness Day on Facebook to see the different ways people acknowledged this special day. Perhaps the most exciting reaction to the day came from Pope Francis. Having a world leader acknowledge September 7 made it all the more powerful. Thank you to Elizabeth Vroom and Nicoletta Madia for organizing this historic event. Can't wait to make September 7, 2015 even bigger!
Run For Our Sons
Star Wars Half Marathon Weekend
There's still time to join our team of Jedis fighting against Duchenne at the Inaugural Star Wars Half Marathon Weekend! We're heading to Disneyland January 16th – 18th and still have entries available for the 5K, 10K and half marathon races. All of our runners receive a 1 day/1 park ticket to Disneyland, a team technical shirt, invitations to our private team pasta dinner, and much more! Fulfill your running destiny...join our team and help us end Duchenne.
Join Us in the Big Easy!
We're heading to New Orleans for the 4th year in a row, and we hope you can join us! Run a fast, flat course through some amazing historic neighborhoods, then celebrate at the post-race party and enjoy some fantastic local music and food on famed Bourbon Street. There's are full marathon, half marathon and half marathon relay distances available. Register now.
Run the California Coast
The Big Sur International Marathon is one of the most breathtaking races in the country, winding along Highway One near Monteray, California, and for the first time ever, Run For Our Sons has entries into this sold-out race! Join our team soon, there's a limited number of entries available!
The Happiest Race on Earth
A huge thank you to the 116 members of our Disneyland Half Marathon Weekend team for making this one of our best races yet! Runners and walkers of all ages, including four boys with Duchenne and one with Becker, participated in the Family Fun Run 5K, the 10K, and Half Marathon, and for the truly ambitious, the Dopey Challenge, which is the 10K and half marathon on back-to-back days! Our team celebrated with Mickey Mouse at a team pasta dinner, reunited with old friends, and made new ones throughout this fun-filled weekend, and in the process raised almost $125,000!! Check out the photos on Facebook, and plan to join us next year for one of our most popular races of the year!
Runner in the Spotlight
This month's runner spotlight is on Ruth Baquiran, who recently ran both the 5K and the 10K - her first races ever! - at the Disneyland Half Marathon Weekend, for her son Nathan who was diagnosed last year. Meet Ruth.
Now recruiting:
Baltimore Running Festival - October 18, 2014
Star Wars Half Marathon Weekend - January 16 – 18, 2015
Chevron Houston Marathon & Aramco Houston Half Marathon - January 18, 2015
Rock 'n' Roll New Orleans Marathon & Half Marathon - January 25, 2015
Big Sur International Marathon - April 26, 2015
Community Corner
For 16 years, the Peterson family along with friends and partners in Rockford, Michigan have organized one of PPMD's most successful and long-running events: Mitchell's Run Thru Rockford. To date they have raised nearly $900,000! It is amazing to see a community come together to end Duchenne year after year. A huge THANK YOU to the Peterson family, Rockford community, and every one of MRTR's awesome runners!
Upcoming Events
• September 20: Ducky Derby
• September 20-21: Rock 'n' Roll Philadelphia Half Marathon & 5K
• September 27: Coach To Cure MD
• September 27: Dash for Derek
• September 27: Garrett's Hero Run
• September 27: Sam Strong Run 2 End Duchenne
• October 4: Sam's Day
• October 11: Argyle Run
• October 12: Bank of America Chicago Marathon
• October 12: GoodLife Fitness Run Victoria Marathon
• October 18: Baltimore Running Festival
• October 18: GR .1K
• October 25: Brookhaven 5K Fun Run/Walk
• October 25: Boo Run
• October 25: Lancaster Run For Our Sons 5K/10K
Check out our full Calendar and sync it with your personal calendar!
Education
The Breath of Life: New 3-Part Pulmonary Webinar Series
The lungs require the strength of many muscles to help a person to breathe and cough. As people with Duchenne grow older, the respiratory muscles, like the skeletal muscles, become weaker. Like the heart, every person with Duchenne will have pulmonary issues of some type. This is such an important area of care and concern that we pulled together pulmonary experts from across the US to address important issues during a webinar series starting September 24. Find out details here.

It's Flu Season Again!

Unfortunately, influenza viruses love the fall weather as much as we do. Everyone needs to be protected against influenza, and protecting children with neuromuscular disease is especially important. Several of you have also asked about the enterovirus, a mild to severe respiratory illness going around now. The CDC has provided helpful information so that you can be aware of the symptoms caused by this virus. Learn more.

People with Disabilities: National Preparedness Month

September is National Preparedness Month, and people with disabilities were the focus of preparedness activities at the Centers for Disease Control and Prevention (CDC). Many people with disabilities and their caregivers do not plan for disasters, or are not included in disaster planning. CDC works to make sure people of all abilities – including those with disabilities – are prepared for an emergency. Read the CDC blog titled Emergency Planning for All Abilities.

Susie's Story

Many of you have followed Susie’s LVAD journey on Facebook. Recently, Susie had a very serious blood clot incident that unfolded online. Susie is recovering wonderfully and shares her story with PPMD so that others can learn from her experience. Read her story.

Social Security Disability Help

If you have a child with Duchenne or Becker muscular dystrophy, you may be eligible to receive funds from the government to help with the burden of expenses brought on by day to day life with muscular dystrophy. Social Security Disability benefits, in the form of Supplemental Security Income (SSI), can help ease the financial stresses of raising a child with this disability so that you can focus on the important things in life, such as caring for and enjoying time with your child. Learn more.
More Education Updates
PPMD convened a pre-conference meeting of physical therapists, occupational therapists, and clinical coordinators who specialize in the research, assessment, longitudinal data collection, and treatment of people with Duchenne. Find out about what was discussed at this meeting and next steps.
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.
Simple Actions
Leverage the United Way

The annual United Way campaigns are getting underway throughout the country. Last year many families had great success leveraging both the annual United Way campaign and Combined Federal Campaign (CFC) to raise money to fight Duchenne. Many people have a tradition of donating to the United Way, are expecting their company campaigns to start, and are looking for good causes to earmark their donations. Download PPMD's United Way/CFC Fundraising Campaigns guide (PDF) which provides information on how local United Way operate and how to work to arrange funding through them.
Coach To Cure MD

Are you looking for more ways to get involved with Coach To Cure MD? We are in need of Duchenne family volunteers to attend the following games on September 27th. If you know any families that may be interested, please contact our team captain, Danielle Garrigan at Danielle@ParentProjectMD.org!
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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© Parent Project Muscular Dystrophy 2014 — All Rights Reserved.

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