This time of year everyone is in holiday mode, or getting into holiday mode, or sick of being in holiday mode. But something else happens this time of year that effects many of us – especially our children. Report cards. The end of the year is when teachers meet with parents to tell them how little Timmy is doing in class. If Timmy is in high school or college, grades come out. But kids aren’t the only ones “graded” this time of year. Often, at our jobs, we undergo year-end reviews. Our bosses assess how we are doing and what our goals are for the coming year.

PPMD prides itself on a full “to do list” that we hope reflects what is most important to you and your family. And we pride ourselves on our transparency. We want you to know where your donations go, what your energies are being put towards, and why you should continue to believe in us and our mission.

Below is a list of what PPMD set out to do in 2011, what we accomplished, and some of the things we are working on for 2012:



  • What we accomplished in 2011...
    • PPMD awarded a $750,000 grant to Dr. Ron Victor and Cedars-Sinai Medical Center for the ongoing Sildenafil/Tadalafil study (Read release).
    • PPMD awarded a $250,000 grant to PTC Therapeutics to advance drug discovery efforts in identifying a new treatment that improves heart function in Duchenne and Becker (Read release).
    • PPMD awarded a $250,000 supplement to Wellstone Centers at University of Pennsylvania, which includes the work of H. Lee Sweeney at UPenn and Stan Nelson at UCLA.
    • PPMD awarded a $150,00 grant to Woody Benson, MD and Cincinnati Children’s Medical Hospital to continue work using cardiac MRI to measure serial changes in myocardial strain track cardiac dysfunction.
    • PPMD awarded a $100,000 grant to National Organization for Rare Disorders (NORD) in support of the ataluren access program.
    • PPMD awarded Tejvir S. Khurana, MD, PhD, Professor of Physiology at the University of Pennsylvania, an End Duchenne Grant for $98,000 (Read release). This bridge funding will allow Dr. Khurana the resources to continue work to upregulate utrophin and correct the underlying defect in mdx muscle with the objective of translation to the clinic.
    • PPMD awarded three separate exploratory grants at $50,000 each to researchers at The Ohio State University and Nationwide Children’s Hospital (Read release). Each grant helps continue work in cardiomyopathy. Heart issues affect all patients with Duchenne.
    • PPMD provided $50,000 in travel assistance grants to AVI for participants in exon skipping trials (Read release).
    • PPMD awarded over $40,000 to SomaLogic as part of a three-way funding agreement that included Cincinnati Children’s Hospital as well, to use SomaLogic’s proprietary screening technology to identify biomarkers in Duchenne (Read release).
    • PPMD announced funding of a three-way collaboration between Nationwide Children’s in Columbus, Children’s National Medical Center in Washington, DC, and TREAT-NMD, designed to prioritize FDA-approved drugs for further testing: Review of Approved Drugs for Duchenne (RADD) Working Group.
    • PPMD partnered with PLoS to launch a new publishing initiative (PLoS Currents: Muscular Dystrophy) to accelerate the pace of muscular dystrophy research through the rapid sharing of new findings and ideas.
  • What we are working on for 2012...
    • PPMD will award Dr. Justin Fallon and Tivorsan Pharmaceuticals a grant of $500,000 to continue moving biglycan towards human clinical trial. PPMD awarded Dr. Fallon the first End Duchenne Grant in 2008 which leveraged $5 million in NIH funding. PPMD is raising funds for this project through the 2011 Year End Campaign.
    • PPMD will continue to proved a $250,000 supplement to Wellstone Centers at University of Pennsylvania, to support the work of H. Lee Sweeney at UPenn and Stan Nelson at UCLA.
    • PPMD will continue grant funding to NORD in support of the ataluren access program.
    • PPMD is currently reviewing over 35 grant applications, including End Duchenne Grant applications, Exploratory/Bridge Applications, PPMD Investigator Applications, and Supplementary Funding Applications. We continue to receive grant applications from the world’s top Duchenne researchers and will always support the most promising therapies.


  • What we accomplished in 2011...
    • PPMD marked ten years of advocacy in Washington, D.C. by holding the One Voice Advocacy Summit (Read release). Over the last decade, PPMD’s efforts in our nation’s capitol have led to more than $200 million in Duchenne-specific federal funding.
    • PPMD published its One Voice Summit report, titled Strategic Directions for the Next Action Plan to End Duchenne.
    • PPMD hosted annual Advocacy Conference in Washington in February.
    • PPMD showed public support of the FDA’s "PDUFA Reauthorization Performance Goals and Procedures, Fiscal Years 2013 Through 2017” and their encouragement of therapies for rare diseases (Read release).
    • PPMD participated in the Rare Disease Congressional Congress (Watch the video).
    • PPMD asked to join National Organization for Rare Disorders Board and the Institute of Medicine’s Committee on Pediatric Studies.
    • PPMD has joined up with other groups in the disability community, in a coalition to halt the CDC’s consolidation of all disability programs funded through the National Center on Birth Defects and Developmental Disabilities. Language was submitted to the subcommittee (Read the release).
    • PPMD submitted a letter to the U.S. Department of Health and Human Services (HHS) weighing in on changes to the regulations overseeing research on human subjects (Read the letter).
  • What we are working on for 2012...
    • Registration for the 2012 Advocacy Conference is underway. We need your voice to join our voice, more than ever before.
    • PPMD continues to have meaningful dialogue with the FDA about rare disease and the drug approval process.
    • PPMD will announce its priorities for advocating on Capitol Hill in early 2012, to include efforts involving, CDC, NIH, DOD, and FDA.
    • I will continue to serve on behalf of PPMD and the Duchenne community, on the NORD Board, IOM Committee, and the of Department of Defense Congressionally directed research program in Duchenne (DMDRP).


  • What we accomplished in 2011...
    • PPMD announced the release of its new, free mobile application (app), putting emergency care information and critical updates at your fingertips.
    • PPMD and DuchenneConnect’s Direct Access Webinar Series, including webinars on exon skipping, DELOS study, transition care: adolescent to adult, Fat Embolism Syndrome, and an upcoming webinar on biglycan.
    • DuchenneConnect launched the Clinic Services Resource available for patients and families of those affected by Duchenne and Becker. The Clinic Services Resource features a web-based directory that will unite patients, families, and healthcare providers to improve healthcare together.
    • PPMD announced that it received a grant from the National Institutes of Neurological Disorders and Stroke (NINDS), part of the National Institutes for Health. This grant was awarded to fund PPMD's ongoing interest in maximizing the clinical trial experience for participating patients with Duchenne. PPMD will survey and interview the Duchenne community regarding the clinical trial experience (Read the release).
  • What we are working on for 2012...
    • PPMD will introduce a newly revamped Care section at The new site will be more interactive and arm you with even more information to care for your son and to share with the other caregivers in his life.
    • PPMD and DuchenneConnect will continue to host monthly webinar as part of the Direct Access Webinar Series, including updates on MRI technology and advocacy.
    • PPMD will continue to update the mobile application (app), with emergency care information.


  • What we accomplished in 2011...
    • PPMD revamped, making it more user friendly and an even more go-to Duchenne resource for the international community.
    • PPMD hosted the Annual Connect Conference in Baltimore with over 75 presenters. A full list of presentations were made available for download.
    • PPMD hosted the third Endocrine Meeting, in Toronto in December, gathering top experts to discuss small stature, delayed puberty, weight management, and bone fragility.
    • PPMD Board Member and Clinical Assistant Professor at Baylor College of Medicine, James T. Poysky, PhD, wrote a companion piece to Education Matters - PPMD's guide to better assist parents and teachers in providing a quality education for the student with Duchenne.
    • PPMD launched HerSelf First: Helping Women Reconnect, Reinvest, Rediscover – a new initiative to promote women’s wellness. Workshops began in a couple of cities around the country.
  • What we are working on for 2012...
    • PPMD will host the Annual Connect Conference in Fort Lauderdale, June 28 – July 1, 2012. More information is available online.
    • PPMD will host a West Coast Connect Meeting in San Diego, April 13 – 15, 2012. Registration information and a list of topics is available online.
    • PPMD will update the full Education Matters series.
    • HerSelf First will hold more workshops around the country in the new year.


  • What we accomplished in 2011...
    • Our online community site and our Facebook and Twitter pages grew and grew, becoming critical resources for the community to share stories, concerns, information, and support.
    • A new FACES group launched in Northern California, and ongoing FACES groups in Arizona and Colorado/Wyoming hosted important meetings for the Duchenne community about care.
    • Run For Our Sons continued to grow, adding new races in Philadelphia and St. Louis, and with 400 runners raising nearly $1 million in 2011.
    • Coach To Cure MD marked its fourth year by hitting the $1 million dollar mark in overall fundraising, with over 500 schools and over 10,000 coaches participating nationwide.
    • Your fantastic grassroots events grew by 33% and were incredibly creative and inventive, including auctions, pizza-thons, wine tastings, 5Ks and family fun runs, and even our first .1K race!
  • What we are working on for 2012...
    • We will continue to update and optimize our online presence so that you can connect with families and friends of the Duchenne community, around the world.
    • We will launch new FACES groups, including Tennessee.
    • Run For Our Sons will grow again adding New Orleans, Little Rock, Cleveland, and Grand Rapids marathons, plus the New York City Triathlon and Conquer the Canyon (a hike through the Grand Canyon).
    • Coach To Cure MD will celebrate five years with some exciting announcements and even more ways for football fans to get involved.
    • Our calendar is already starting to fill with more grassroots events. We are so appreciative of all of you who roll up your sleeves and get your community involved in raising awareness and funds to fight to end Duchenne.

We are grateful for the incredible minds that are seeking treatments to give our sons longer, healthier lives.
We are grateful to the eloquent voices that speak on behalf of our sons to tell the world about Duchenne.
We are especially grateful to the generous hearts that embrace our sons and join us in the fight to end Duchenne.

With so many important organizations asking for your hard earned money, we hope that you will look at what we’ve done this year and our goals for next year, and include supporting PPMD in your list of priorities.

We see a future without Duchenne, and we WILL make it happen.


Pat Furlong, Founding President, CEO
Read more PPMD Staff Blogs

Views: 3430


You need to be a member of PPMD Community to add comments!

Join PPMD Community

Need help using this community site? Visit Ning's Help Page.



© 2023   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service