by: Parent Project Muscular Dystrophy
2019 PPMD Annual Conference Resources & Recordings
JUNE 27 – JUNE 29, 2019 | Orlando, Florida
Thank you doesn’t begin to express our gratitude to everyone who participated in our 25th Annual Conference in Orlando, Florida! It was a wonderful opportunity to reconnect with old friends and make new ones. We learned the latest updates in research from both scientists and industry, as well as critical care information. And our adult Duchenne community took center stage to share their experiences, their stories.
If you weren’t able to attend in person or watch our live stream, or if there’s something you want to hear again, you can now download and watch most of the general sessions and our first-ever Town Hall via the links below.
As we enter PPMD’s 25th Anniversary year and continue to lead the fight against Duchenne from every angle, we are committed to investing more than ever before in significant projects across our pillars of Research, Care, Education and Advocacy. With each new idea, and our aggressive pursuit of every promising opportunity, PPMD needs the support of our committed donors. As we recognize 25 years of leading the fight to end Duchenne, please consider making a contribution to help PPMD continue to fight at parentprojectmd.org/25.
Join us next June when we return to Scottsdale, Arizona for our 26th Annual Conference!
Understanding Duchenne from the Start – Part 1
- Introduction: So It Begins
Pat Furlong
Download PDF - Making a Care Plan & Lining up Your Team
Christina Trout, RN, MSN
Download PDF - Why Physical Therapy Is so Necessary
Claudia Senesac, PT, PhD, PCS
Download PDF - Diet & Duchenne: Simple Nutrition Advice
Kent Williams, MD
Download PDF
Understanding Duchenne from the Start – Part 2 (Behavioral Challenges)
- Talking to School, Family, & Children about Duchenne
James Poysky, PhD
Download PDF - Early Identification of Boys with Developmental & Socio-Emotional Delays
Amy Wagner, PhD, MAEd
Download PDF - Navigating Challenges at School & Home
Denise Gruender, BS
Download PDF
Where We Have Come From/Where We Are Going
Pat Furlong
The Complexities of Duchenne
Moderator: Pat Furlong
- Duchenne: A Multisystem Disease
Stan Nelson, MD, PhD
Download PDF - Variability: Modifiers to the Gene
Kevin Flanigan, MD
Download PDF - Measuring Outcomes in the Face of Variability
Cliff Bechtold
Download PDF - Challenges in Designing Trials
Craig McDonald, MD
Download PDF
Addressing the Complexities of Duchenne – What You Should Know
Moderator: Anessa Fehsenfeld
- Attacking Duchenne from Every Angle
Anessa Fehsenfeld, PPMD Chairman
Download PDF - Duchenne Research and Clinical Trials
Abby Bronson
Download PDF - Working to Facilitate a Favorable Access Environment
Annie Kennedy
Download PDF - Improvements in Care: Extending Quality of Life with a Focus on the Future
Kathi Kinnett, MSN, APRN
Download PDF - The Patient Voice: Your Data Informing the Drug Development Ecosystem
Ryan Fischer
Download PDF
Quality of Life and Emotional Health of the Family: A Panel Discussion
Moderator: Erik Henricson, PhD
Panel: James Poysky, PhD; Rachel Poysky; Denise Gruender, BS; Jamie Jackson, PhD; Jessica Rownd
Download PDF
Our Stories Matter (Lance Hester)
Lance Hester
Gene Therapy: What We Know Today & What We Will Need to Know Tomorrow
Moderator: Tim Cripe, MD, PhD
- Understanding Gene Therapy – What We Know Today
Barry Byrne MD, PhD
Download PDF - Panel Discussion: Current Hurdles in Gene Therapy
Moderator: Tim Cripe, MD, PhD
Panel: Lee Sweeney, PhD; Kevin Flanigan, MD; Kathryn Wagner, MD; Barry Byrne, MD, PhD
Download PDF
Gene Therapy Trials
Moderator: Richard Klein
- Solid
Genevieve A. Laforet, MD, PhD
Download PDF - Pfizer
Michael Binks, MD
Download PDF
News Release from Pfizer - Sarepta
Doug Ingram
Download PDF
Downstream
Moderator: Lance Hester
- Roche/Genentech
Roxana Dreghici, MD
Download PDF - Santhera
Kristina Nygren, MD
Download PDF - FibroGen
Bassem Elmankabadi, MD
Download PDF
News Release from FibroGen
Our Stories Matter (Stephen Marchesani)
Stephen Marchesani
Duchenne & the Heart
Moderator: Abby Bronson
- Introduction & Overview
Linda Cripe, MD
Download PDF - Outcome Measures for the Heart
Kan Hor, MD
Download PDF - Second Generation Micro-Dystrophin
Dongsheng Duan, PhD
Download PDF - Understanding Gene Therapy for the Heart
Lee Sweeney, PhD
Download PDF - Cumberland Pharmaceuticals
Larry Markham, MD
Download PDF
Our Stories Matter (Stacie Al-Chokhachi)
Stacie Al-Chokhachi
Inflammation and Fibrosis
Moderator: Abby Bronson
- Catabasis
Joanne Donovan, MD, PhD
Download PDF
News Release from Catabasis - PTC
Brian Pfister, PhD
Download PDF - Italfarmaco
Paolo Bettica, MD, PhD
Download PDF - ReveraGen
Paula Clemmons, MD
Download PDF - Mallinckrodt
Jacqueline Delfgaauw, PhD
Download PDF
Our Stories Matter (Buddy Cassidy)
PAAC Member – Buddy Cassidy
CRISPR – Future Strategies in Gene Therapy
Moderator: Buddy Cassidy
- Pre-Clinical Research in CRISPR
Melissa Spencer, PhD
Download PDF - The Path to the Clinic
Charles Gersbach, PhD
Download PDF
Understanding Immunity*
Moderator: Sharon Hesterlee
- Innate and Acquired Immunity
Carrie Miceli, MD, PhD
Download PDF
Gene Correction
Moderator: Richard Klein
- PTC
Richard Able Jr., PhD
Download PDF - Sarepta
Gilmore O’Neill, M.B., M.M.SC.
Download PDF - Wave
Michael Panzara, MD, MPH
Download PDF
News Release from Wave - NS Pharma
Leslie Magnus, MD
Download PDF
Our Voice Matters: Duchenne Town Hall Forum
Moderator: Pat Furlong
Hands-On PT Sessions
Claudia Senesac, PT, PhD, PCS
Download PDF
* The presentation entitled “Overcoming Neutralizing Antibodies for Broader Inclusion” (Jude Samulski, PhD) could not be archived due to concerns over confidential information that was shared. However, PPMD knows that there are numerous questions around antibodies as an inclusion criteria in gene therapy trials and will be addressing this topic in our upcoming Gene Therapy Webinar Series launching late summer/early fall.
