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Raising Over Voices for Every Single One
February 2017
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Every year at this time, families from across the country gather in Washington, D.C. to represent the Duchenne community on Capitol Hill. It is one of the most inspiring few days of the year for me and PPMD, seeing the coming together of so many different people with so many different experiences, but one unified goal to end Duchenne.

And it's not just parents. It's not just young adults with Duchenne. It's siblings and aunts & uncles and neighbors and colleagues. People who realize that adding their voice to our community's message on behalf of those who aren't able to attend is one of our greatest weapons in this fight. For two decades we have been sharing our stories in D.C. We have some wonderful champions, representing us and the thousands of other rare diseases in everything they do. And this year we have made new friends, new champions, as a new administration takes office and new members join Congress.

Not everyone can make it to our Advocacy Conference, we get that. But there is still plenty you can do from your computer and your phone. Make sure you are signed up for Action Alerts from PPMD and encourage your family and friends to do the same. It's amazing what a 45 second email can do!

It seems like the headlines in our community change every day sometimes hourly. But one thing we can do consistently as a community is make sure leadership in D.C. knows what Duchenne is and understands how critical their support is. We are each other's voice when we advocate, whether in one-on-one meetings with our Members or via email and phone calls.

Thank you for using your voice to help end Duchenne for every single one.

Warmly,


Pat Furlong
PPMD Founding President & CEO


Research
Deflazacort Approved in the U.S. for Duchenne
On February 9, the FDA approved EMFLAZA™ (deflazacort) for Duchenne. This is the first approval of a steroid for Duchenne and a therapy that applies to people with Duchenne 5 years of age and older, regardless of genetic mutation. Representatives from Marathon Pharmaceuticals joined us for a webinar to provide information about EMFLAZA's FDA-approved label, patient support services, access to this medication, and to respond to questions from our community. On February 13, during the 2017 Advocacy Conference special Forum on Access to Emerging Therapies, Marathon announced that they would be pausing the commercialization of EMFLAZA. This pause will allow Marathon to consider the feedback they have received from the community since last week's announcement regarding pricing and potential for combining this therapy with other approved drugs in the future. Marathon has assured families that during this pause, access to deflazacort will not be disrupted. EMFLAZA's website can help you navigate questions you may have.
PPMD Awards $600,000 Grant to NJIT & Talem for Next Phase of Upper Extremity Exoskeleton
PPMD is proud to announce a $600,000 grant to the New Jersey Institute of Technology (NJIT) and Talem Technologies as part of our ongoing exploration of robotic technology to assist people living with Duchenne. We have always supported innovation, and believe this exoskeleton technology has the ability to spare muscle deterioration keeping people with Duchenne stronger for longer, as well as the potential to assist everyone living with Duchenne. If you are a non-ambulatory member of our community, please consider whether participation in this research effort might be right for you. Learn more.
PPMD Awards $250,000 Grant to UT Southwestern Medical Center to Explore CRISPR/Cas9 Technology
We could not be more grateful to every single person in the Duchenne community who donated to PPMD's holiday campaign to make this grant possible! This grant, part of PPMD's Gene Transfer Initiative, will support Dr. Olson's ongoing study of CRISPR/Cas9 technology as a potential treatment for Duchenne. Thanks to your support, we will find out just what CRISPR/Cas9 can do–and will continue to invest in new, innovative research until we end Duchenne for every person, for every family. Learn more about Dr. Olson's study.
PPMD's Duchenne Drug Development Roundtable: Bringing Industry Together to Accelerate Research
February 1 was the first of PPMD's Duchenne Drug Development Roundtable (DDDR) three-meeting series to address clinical trial challenges and opportunities within our Duchenne space. The DDDR is a group of committed innovators representing iIndustry and relevant stakeholders that has the goal of accelerating the development of treatments for Duchenne through open discussion. It is our hope that this DDDR meeting series will assist PPMD and the Duchenne community to work together to drive forward collaborations and projects that will benefit all partners working in this space – and yield effective therapies that are accessible to all who need them. Learn more.
[Webinar Recording] Catabasis Provides Update
The Catabasis team joined us last week to discuss what they have learned from Part B of the MoveDMDŽ trial of edasalonexent (CAT-1004) in Duchenne and what comes next in the MoveDMD program. You can watch the recording of the webinar by clicking here.
[Webinar Recording] Mission DMD: FibroGen's Anti-Fibrosis Program
FibroGen joined us for a webinar discussion on the company's investigational drug Pamrevlumab (FG-3019), which is an anti-CTGF monoclonal antibody. Speakers provided an overview of the rationale for evaluating Pamrevlumab in Duchenne patients, as well as a description of the recent protocol amendment for this ongoing clinical trial in non-ambulatory boys with Duchenne. You can watch the recording by clicking here.
[Webinar Recording] Vamorolone (VBP15) Clinical Trials in Duchenne
ReveraGen BioPharma CEO Dr. Eric Hoffman joined us for a webinar discussion on vamorolone (VBP15) clinical trials in Duchenne. Dr. Hoffman presented a brief history of the vamorolone program, explained current and anticipated clinical trials, and answered questions from the community. You can watch the recording by clicking here.
[Webinar Recording] Summit's Utrophin Modulation Program
Summit joined us for an educational webinar on utrophin modulation, an approach for Duchenne that has the potential to treat all patients. Our speakers discussed the basic biology of utrophin, how the company is working to understand and characterize the activity of utrophin modulators through biomarkers in muscle biopsies, and a brief overview of Summit's current Phase 2 clinical trial, PhaseOut DMD, which is ongoing in the US and UK. You can watch the recording of the webinar by clicking here.
Other Research News
Catabasis Announces Results for Part B of the MoveDMD trial for Edasalonexent (CAT-1004) in Duchenne
PPMD Awards $2.2 Million Grant to Explore Gene Therapy in Duchenne
[Webinar] A Closer Look at the Potential of CRISPR/Cas9 in Duchenne
Trial Finder/Antidote
PTC Provides Update on Next Steps for Translarna
Akashi Therapeutics Provides Update on Three Duchenne Compounds
Advocacy
ACTION ALERT: Urge Congress to Protect Duchenne Community's Healthcare Priorities
This week, PPMD advocates from across the country are storming Capitol Hill to ensure legislators make Duchenne and rare disease a priority. This year's agenda includes urging Congress NOT to repeal the Patient Protection and Affordable Care Act until there is a guarantee that people with Duchenne will continue to have access to equal or better coverage. Please take a moment today to tell Congress why this matters so much to our community!
PPMD Hosts Forum on Access to Emerging Therapies
Following decades of strategic advocacy, research investment, and biopharmaceutical development, the U.S. Duchenne community recently arrived in the post-approval space and is poised to receive additional novel therapies from a robust innovation pipeline. PPMD is engaged on behalf of families nationwide as industry sponsors work with the FDA to navigate the critical regulatory steps. We are also working now within the access environment to help ensure that families have timely access to these disease-modifying medications. It is our hope that this Forum will be an important step in helping to continue to empower our Duchenne community and our partners to navigate the regulatory and approval pathways necessary to achieve desired levels of clinical treatments and care appropriate for themselves/their children. Learn more about this week's Forum in D.C.
PPMD Participates in Newborn Screening Committee Meeting
Earlier this month, PPMD's SVP of Legislation & Public Policy, Annie Kennedy participated in the Secretary's Advisory Committee on Heritable Disorders in Newborns and Children in our ongoing effort to have Duchenne part of newborn screening procedures. Click here to read Annie's testimony to the committee. We have also joined dozens of other organizations to ensure newborn screening receives federal funding. To that end, PPMD recently signed on to a Newborn Screening Funding Letter – read the full letter here.
Other Advocacy News
PPMD Signs on to Defense Health Research Consortium Letter to House and Senate Leadership to ensure funding from the 2017 Defense Appropriations Act
Meet the 2017 PAAC
Pat Furlong Joins National Health Council Board
Community
Run For Our Sons
Walt Disney World Marathon Weekend 2018
Registration is now open for our 2018 Walt Disney World Marathon Weekend team, and our entries are filling up fast! Join us for Run For Our Son's biggest and best race weekend of the year, where more than 180 runner and their families head to the most magical place on Earth for a weekend of races, team activities, and of course, hanging out with Mickey and friends! This year will be the 25th anniversary of the Full Marathon, so participants of that race will receive an extra special commemorative medal. Sign up today and join us January 3-7, 2018!
TCS New York City Marathon
The TCS New York City Marathon is the largest marathon the world, and one that is unmatched in energy and excitement! Run For Our Sons has a limited number of entries available, and we'd love for you to join us! The city's five boroughs transform into the world's largest cheering zone on November 5 as runners take to the streets of the Big Apple, and you can be part of it all. Fill out our interest survey to get started.
Rock 'n' Roll Seattle Marathon & Half Marathon
Run For Our Sons is thrilled to be a charity partner in this year's Rock 'n' Roll Seattle Marathon & Half Marathon on June 18. This popular race normally sells out, but we will have post-sell out entries! Join us for this challenging and scenic course through the beautiful city of Seattle and its surrounding suburbs. Grab some friends, form a team, and go the distance to end Duchenne in Seattle!
Runner in the Spotlight
Dave Friedman joined the Run For Our Sons team at the Walt Disney World Marathon Weekend in 2009, three years after his nephew Danny was diagnosed. Since then, he has run at Disney World seven times, as well as participated in countless other races across the country to share Danny's story and raise awareness and funds to end Duchenne. Meet Dave.
Now recruiting:
Rock 'n' Roll Raleigh Marathon, Half Marathon & 5K - April 1, 2017
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
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
• February 18, 2017: UF PT Golf Tournament
• February 18, 2017: Ice Duchenne
• March 4, 2017: Shake It to End Duchenne
• 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
Check out our full Calendar and sync it with your personal calendar!
Education
PPMD Designates Yale New Haven Children's Hospital a Certified Duchenne Care Center
PPMD is excited to announce our 13th Certified Duchenne Care Center (CDCC) the Yale New Haven Children's Hospital's Muscular Dystrophy Program! The Pediatric Muscular Dystrophy Program at YNHCH is co-directed by Brian Smith, MD and Cristian Ionita, MD. Dr. Ionita was recently recruited from Seattle Children's Hospital (also a CDCC), and is joining a compassionate team of experienced Duchenne providers. We are thrilled to award the YNHCH team certification for their dedication to Duchenne patients meet the clinic!
Every Single One Tour - L.A. Recap
In late January, families from across California gathered together at Children's Hospital Los Angeles (CHLA) - one of PPMD's Certified Duchenne Care Centers - to hear the latest updates in Duchenne research, advocacy, and care. Thank you to all of our presenters, the extraordinary clinical providers at CHLA, and our amazing families who joined us for an information-packed day. Click here for a full wrap-up. Next stops... Seattle, WA and Iowa City, IA!
More Education Updates
SAVE THE DATE! PPMD's 2017 Calendar
What You Need to Know about Duchenne & Viral Gastroenteritis
Simple Actions
New Research & Clinical Trial Tools on PPMD's DuchenneConnect!
We are excited to announce three new tools now available on PPMD's DuchenneConnect! These tools are meant to help you in your journey as you gather information around potential research studies and potential new exon skipping therapies. Learn more.
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
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Run For Our Sons
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© Parent Project Muscular Dystrophy 2017 – All Rights Reserved.

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