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2013 Connect Conference Presentations

2013 Connect Conference Presentations

The theme of the 2013 Connect Conference was Better, Faster, Now. This is how we categorize projects in our research portfolio. But actually, Better, Faster, Now, goes beyond research alone. Just like PPMD.

Please right-click on a link to save presentation PDF and MP3 files to your computer.

Thursday: BETTER

Making Life Better... Knowing more, being educated, knowing what to look for, what to ask, and where to go.

  • Duchenne: Life Lessons on Taking the Sting of out the Diagnosis – PDF - MP3
    Pat Furlong, PPMD President & CEO

  • Six Important Areas of Care – PDF - MP3
    James Dowling, MD, PhD

  • The Importance of Bone Health, Endocrine, Oral Health, GI, and GU – PDF - MP3
    Garey Noritz, MD

  • Report: Transition Care Meeting – PDF - MP3
    Kathi Kinnett, MSN, CNP

  • Parents and Clinicians Weigh In: Making the Clinical Trial Process Better – PDF - MP3
    Holly Peay, MS
  • Encouraging Independence: A Panel Discussion – MP3
    Max Gravenstein, Patrick Denger, Ben Dupree, Tayius Surampudi, Jonathan Piacentino, Ben Cumbo

  • Living Duchenne: Life Lessons for Every Age and Stage – MP3
    Rachel Poysky, Ivy Scherbarth, Jill Castle, Ellen Wagner

Friday: FASTER

Moving Faster... Understanding research, advocacy and elements that will help move the field faster.

  • Our Lives with Duchenne – PDF - MP3
    Ivy Scherbarth

  • Keynote Address: The Medicinal Value of Pleasure and Touch – PDF - MP3
    Evelyn Resh, CNM, MPH

  • Research with a Mission – PDF - MP3
    Sharon Hesterlee, PhD

  • Beyond 51: Rare Deletions, Duplications, and Multi-Exon Skipping – PDF - MP3
    Toshifumi Yokota, PhD; Steve Wilton, PhD; Adeline Vulin-Chaffiol, PhD
  • Natural History and MRI as a Biomarker – PDF - MP3
    Krista Vandenborne, PhD
  • Department of Defense Duchenne Funding – PDF - MP3
    Marielena McGuire, PhD

  • Putting Patients First: The White Paper – PDF - MP3
    Julianna Belelieu
  • Benefit/Risk: What We Have Learned – PDF - MP3
    Holly Peay, MS; John Bridges, PhD
  • Advocacy: Our Weapon in the Fight to End Duchenne – PDF - MP3
    Ryan Fischer

  • FDA Perspective – PDF - MP3
    Robert Temple, MD

  • Laying the Groundwork for GalNac Transferase Gene Therapy – PDF - MP3
    Paul Martin, PhD

  • Biomarker Collaboration with Somalogic – PDF - MP3
    Sally Nelson, PhD
  • Beyond Steroids: Blocking Inflammation in Duchenne – PDF - MP3
    Denis Guttridge, PhD

  • PPMD’s Pre-Clinical Efforts and the Case for Tadalafil – PDF - MP3
    Lee Sweeney, PhD

Saturday: NOW

What is Now... Discussing what is happening now, and how it impacts the Duchenne community.

  • Mitchell’s Journey and Our Mission – MP3
    Chris Jones


  • Current Issues and Developments in Duchenne Cardiac Care – MP3
    Charles E. Canter, MD PDF; Naomi Kertesz, MD PDF; W. Reid Thompson, MD PDF; David Morales, MD PDF
  • What to Know in an Emergency – PDF - MP3
    Doug Biggar, MD

  • The Trouble with Oxygen – PDF - MP3
    Jonathan Finder, MD
  • Genetics and Proteomics: DuchenneConnect Registry, Biomarkers, Modifiers – PDF - MP3
    Stan Nelson, MD, PhD
  • Where Are We Now: Clinical Trial Updates Introduction – PDF - MP3
    Sharon Hesterlee, PhD
  • Follistatin Gene Therapy – PDF - MP3
    Jerry Mendell, MD
  • Reveragen (VBP-15) – PDF - MP3
    Erica Reeves, PhD
  • PTC Therapeutics (Ataluren) – Unavailable
    Robert Spiegel, MD

  • Results of REVERSE-DBMD (Revatio™) Study – Unavailable
    Kathryn Wagner, MD, PhD
  • Prosensa – PDF - MP3
    Giles Champion, PhD

  • GSK (Drisapersen)  – Unavailable
    Carlo Russo, MD
  • Sarepta (Eteplirsen) – PDF - MP3
    Chris Garabedian
  • Halo (HT-100) – PDF - MP3
    Diana Escolar, MD

  • For DMD Study (Corticosteroids) – Unavailable
    Kathryn Wagner, MD
  • Pfizer (Anti-GDF-8 Antibody) – Unavailable
    Carl Morris, PhD

  • Summit (SMT-C1100)  – PDF - MP3
    Jon Tinsley, MD
  • Lilly (Cialis™)  – PDF
    Ron Victor, MD
  • Tivorsan (Biglycan) – PDF - MP3
    Joel Brausnstein, MD

  • Catabasis (CAT-1000) – PDF - MP3
    Michael Jirousek, PhD
  • 30 Meters Matter™ : What Natural History Data is Showing Us, and Why it is Critical to Clinical Trials – Unavailable
    Craig McDonald, MD (via Skype)
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