Direct Access Webinars

DuchenneConnect and Parent Project Muscular Dystrophy present a webinar series that allows you direct access to experts in the fight to end Duchenne. Whether it's a researcher offering updates on a developing therapy, or industry providing clinical trial information, or experts in care and management, this series will be your resource to find out what is going on in the community from the people making it happen. Please contact us at This email address is being protected from spambots. You need JavaScript enabled to view it. if you have questions about recent webinars or recommendations for upcoming webinars.

Webinar Archive

June 7, 2017

Saprepta's ESSENCE Clinical Trial for Exon 45 & 53 Skipping

Speaker: Dr. Genevieve Laforet, Medical Director - Sarepta

May 17, 2017

Nationwide Children's Hospital Carrier Study

Speakers: Dr. Kan Hor and Dr. May Ling Mah - Nationwide Children's Hospital

April 26, 2017

Givinostat: An HDAC Inhibitor in Development for the Treatment of Duchenne

Speaker:  Dr. Paolo Bettica, VP of R&D - Italfarmaco

March 27, 2017

Akashi Therapeutics:  Update on HT-100

Speaker:  Dr. Marc Blaustein, CEO - Akashi

March 9, 2017

Santhera:  Idebenone and the SIDEROS Trial

Speakers:  Dr. Thomas Meier and Jodi Wolff, PhD, Santhera Pharmaceuticals

February 22, 2017

Updates on NS Pharma's Exon 53 Skipping Trial

Speakers:  Dr. Paula Clemens and Lauren Morgenroth, CINRG

February 2, 2017

Updates on MoveDMD Trial from Catabasis

Speaker:  Dr. Joanne Donovan, CMO - Catabasis

February 1, 2017

MissionDMD: FibroGen's AntiFibrosis Program

Speaker:  Ewa Glossary Link Carrier, MD - FibroGen

January 25, 2017

Summit's Utrophin Modulation: A Universal Treatment Approach to DMD

Speakers:  Jon Tinsley (CSO) and Ralf Rosskamp (CMO) - Summit Therapeutics

January 18, 2017

Vamorolone (VBP15) Clinical Trials in Duchenne

Speaker:  Dr. Eric Hoffman, CEO - ReveraGen BioPharma

December 15, 2016

A Closer Look at the Potential of CRISPR/Cas9 in Duchenne

Speaker:  Dr. Eric Olson, UT Southwestern Medical Center

November 16, 2016 

NS Pharma's Exon 53 Skipping Program

Speakers:  Dr. Paula Clemens and Lauren Morgenroth, CINRG

November 14, 2016

Deflazacort Access: Navigating Potential Changes to Your Prescription

Speakers:  Masters Pharmaceuticals

August 1, 2016

PTC Therapeutics:  Regulatory Update on Translarna

Speaker:  Dr. Craig McDonald, UC-Davis

July 13, 2016

What is CRISPR/Cas9?

Speaker:  Dr. Eric Olson, UT Southwestern Medical Center - Dallas

June 8, 2016

Catabasis Webinar on the MoveDMD Clinical Trial

Speaker:  Dr. Joanne Donovan, CMO, Catabasis Pharmaceuticals

May 18, 2016

FibroGen MissionDMD Webinar

Speakers: Pat Furlong, CEO, PPMD and Ewa Carrier, MD, FibroGen

March 30, 2016

BMS Anti-Myostatin Adnectin Webinar

Speaker:  Leslie Jacobsen, MD, Bristol-Myers Squibb

January 13, 2016

HOPE-Duchenne Trial - Capricor Therapeutics

Speakers:  Deborah Ascheim, MD & CMO, and Linda Marban, PhD & CEO, Capricor Therapeutics

December 16, 2015

Understanding the Potential of Epicatechin

Speaker:  George Schreiner, MD, PhD - President and CSO of Cardero Therapeutics

November 11, 2015

Understanding Myostatin Inhibition

Speakers:  H. Lee Sweeney, PhD - University of Florida, and John Porter, PhD - PPMD

November 4, 2015

Decode Duchenne Genetic Testing Program

Speakers:  Stanley Nelson, MD - UCLA, and Ann Martin and Ann Lucas - PPMD Genetic Counselors

September 16, 2015

Patient-Reported Outcome Measures

Speaker:  Cindy Nowinski, MD, PhD

June 10, 2015

PPMD Webinar on Caregiver Wellness 

Speaker:  Amy Napoli, RDH

June 3, 2015

Pfizer Webinar on Anti-Myostatin (PF-06252616)

Speaker:  Dr. Michael Binks, VP and Head of Clinical Research for Pfizer's Rare Disease Research Unit

May 13, 2015

Catabasis Webinar - Upcoming MoveDMD Phase 1/2 Clinical Trial with CAT-1004 in Boys with Duchenne

Speaker:  Dr. Joanne Donovan, Chief Medical Officer at Catabasis Pharmaceuticals

April 15, 2015

FOR-DMD Study Webinar

Speakers:  Dr. Robert Griggs - University of Rochester Medical Center 

March 31, 2015

PPMD Orthopedic Interventions Webinar

Speakers:  Dr. James McCarthy - Cincinnati Children's Hospital; Terri Cary, PT - Children's Hospital Colorado; Dr. Sue Apkon - Seattle Children's Hospital

March 3, 2015

Dystrophin 101 Webinar: Everything You Always Wanted to Know About the Duchenne Protein

Speakers:  Professor Francesco Muntoni, University College of London, and John Porter, PhD & CEO, PPMD

December 17, 2014


Combination Therapies for Duchenne

Speakers:  Sharon Hesterlee, PhD, Lee Sweeney, PhD, and Dominic Wells, PhD

November 19, 2014


PPMD Pulmonary Care Webinar Series - Adult Pulmonary Care

Speakers:  Dr. Daniel Sheehan and Dr. Lisa Wolfe

October 29, 2014


PPMD Pulmonary Care Webinar Series - Middle Pulmonary Care (Tweens and Teens)

Speakers:  Dr. Jonathan Finder and Dr. Oren Kupfer

October 15, 2014


PPMD Webinar with Santhera Pharmaceuticals - Efficacy of Catena®/Raxone® (Idebenone) on Respiratory Outcome

Prof. Gunnar Buyse (University Hospitals Leuven, Belgium), PI of the study, and Thomas Meier PhD, CEO of Santhera Pharmaceuticals

October 2, 2014


UPPMD Webinar with Prosensa:  Update on Drisapersen and Other Exon Skipping Compounds

Speakers:  Annemieke Aartsma-Rus PhD, Thomas Voit MD, and Brenda Wong MD

September 24, 2014


PPMD Pulmonary Care Webinar Series - Early Pulmonary Care

Speakers:  Dr. Hemant Sawnani, Dr. Richard Shell, and Dr, Oscar Henry "Hank" Mayer

June 5, 2014


Summit Webinar:  Positive Results from Phase 1b Clinical Trial of SMT C1100, a Utrophin Modulator (HOSTED BY ACTION DUCHENNE)

Speaker:  Glyn Edwards, CEO of Summit plc

June 3, 2014


PTC Therapeutics Webinar:  Ataluren (Translarna) Update (HOSTED BY UPPMD)

Speaker:  Dr. Robert Spiegel, Chief Medical Officer at PTC Therapeutics

May 13, 2014


Sarepta Webinar:  Exon Skipping Program Update

Speakers:  Chris Garabedian, President and CEO, and Dr. Edward Kaye, Chief Medical Officer at Sarepta

March 25, 2014


Prosensa Webinar:  Status Update on Drispersen and Follow-On Compounds (HOSTED BY UPPMD)

Speaker:  Giles Campion, MD, Chief Medical Officer at Prosensa

February 5, 2014


PTC Therapeutics:  Update on the ACT DMD Clinical Trial (Ataluren)

Speaker:  Dr. Robert Spiegel, Chief Medical Officer at PTC Therapeutics

November 19, 2013


Update on the Duchenne Natural History Study

Speaker:  Craig McDonald, MD from UC-Davis

October 17, 2013


Sarepta Therapeutics Webinar:  Update on Sarepta's Duchenne Development Program

Speaker:  Chris Garabedian, CEO

                                    August 13, 2013

Prosensa Webinar:  Exon Skipping Program for Duchenne

Speaker:  Dr. Giles Campion, Chief Medical Officer at Prosensa

June 12, 2013

Benefit/Risk in Duchenne Therapies

Speaker:  Holly Peay - VP of Education and Outreach, PPMD

May 6, 2013

Drisapersen Update with GSK (This webinar was not recorded, but a written summary is available.)

Speaker:  John Kraus

March 26, 2013

MD STARnet Revealed

Speaker:  Dennis J. Matthews, MD - Children's Hospital Colorado

March 6, 2013

Cardiac Webinar Part 4: Care for Adults Living with Duchenne

Speakers: Dr. Beth McNally – University of Chicago; Dr. Reid Thompson – Johns Hopkins Children's Center

January 24, 2013

Cardiac Webinar Part 3: The Use of Ventricular Assist Devices (VAD) in Duchenne

Speakers: Dr. Pirooz Eghtesady - Cardiothoracic Surgery, St. Louis Children’s Hospital; Dr. David Morales - Chief of Cardiovascular Surgery, Cincinnati Children's Hosptial Medical Center; Dr. Robert “Jake” Jaquiss - Chief of Pediatric Cardiothoracic Surgery, Duke University; Dr. Patrick McConnell - Cardiothoracic Surgery, Nationwide Children’s Hospital

January 17, 2013

Ataluren Update with PTC Therapeutics

Speaker: Dr. Jay Barth, Vice President for Clinical Development at PTC Therapeutics

December 11, 2012


Summit plc: Phase 1 Clinical Trial results on SMT C1100 (utrophin upregulator program)

Speakers: Jon Tinsley, Senior Director of R&D and Richard Pye, Head of Communications at Summit

November 28, 2012


Cardiac Webinar Part 2: Cardiac Interventions

Speakers: Dr. Larry Markham (Children’s Hospital Vanderbilt, Vanderbilt, TN)and Dr. John Lynn Jefferies (Cincinnati Children's Hosptial, Cincinnati, OH)

October 23, 2012


Sarepta Webinar: Review of the 48 Week Results from the Eteplirsen Phase 2b Extension Trial

Speakers: Chris Garabedian, President and CEO of Sarepta Therapeutics

September 26, 2012


Cardiac Webinar Part 1: Monitoring Heart Disease in Duchenne Boys and Carrier Moms/Daughters

Speakers: Dr. Linda Cripe (Nationwide Children’s Hospital, Columbus, OH) and Dr. Kan Hor (Cincinnati Children's Hosptial, Cincinnati, OH)

August 30, 2012


Back to School Webinar (IEPs and Assistive Technology)

Speakers: Brian Denger and Jill Castle of PPMD

June 6, 2012


New Approaches to Treating Inflammation in Duchenne

Speaker: Dr. Michael Jirousek from Catabasis Pharma

(Unfortunately, we were not able to record this webinar.)

April 25, 2012


Review of the DuchenneConnect Data

Speaker: Dr. Stanley Nelson from the David Geffen School of Medicine at UCLA

March 14, 2012


ImagingDMD Study: Magnetic Resonance Imaging (MRI) as a Tool to Monitor Disease Progression in Duchenne and to Serve as an Outcome Measure in Clinical Trials

Speakers: Dr. Krista Vandenborne and Claudia Senesac, PT, PhD, PCS from the University of Florida

February 22, 2012


An Update from GlaxoSmithKline (GSK) on their Research & Clinical Trials involving Exon 51 Skipping for DMD

Speaker: Dr. Padraig Wright from GSK

(Unfortunately, we were not able to record this webinar.)

January 25, 2012


Parent Project Muscular Dystrophy Advocacy Webinar: The Prescription Drug User Fee Act (PDUFA)

Speaker: Dr. Timothy Franson of FaegreBD Consulting

December 20, 2011


Update on Biglycan

Speaker: Dr. Justin Fallon of Brown University and Tivorsan Pharmaceuticals

November 30, 2011

Parent Project Muscular Dystrophy Webinar: Fat Embolism Syndrome Following Mild Trauma in Duchenne

Speaker: Dr. Laura McAdam from Holland Bloorview Kids Rehabilitation Hospital in Toronto, Ontario, Canada

October 26, 2011

Updates in Genetic Testing

Speakers: Madhuri Hegde, PhD, Senior Director, Emory Genetics Laboratory; Susan Sparks, MD, PhD, Geneticist, Carolinas Medical Center; and Jodi Wolff, MSSW, Director of Clinical Services at the MDA

To read the Q&A from the October webinar, please click here.

September 28, 2011

Transition to Adult Care and the REVERSE-DBMD Clinical Trial (Revatio/Sildenafil)

Speaker: Dr. Kathryn Wagner, Director of the Center for Genetic Muscle Disorders of the Kennedy Krieger Institute and John Hopkins School of Medicine

Webinar Archive