September 9, 2021

DMD: Charting the Path for New Therapeutics and Better Care

Date and time

September 9, 2021 ~ September 9, 2021

8am - 10:30am PDT | 7pm - 9:30 pm PDT

11am - 1:30pm EDT | 10pm - 12:30am EDT

Dear colleagues and friends,


Thank you for participating in our global webinar, “Duchenne Muscular Dystrophy: Charting the Path for New Therapeutics and Better Care,” which was co-organized with Parent Project Muscular Dystrophy (PPMD) and CureDuchenne. We are delighted that more than 1500 registered from 36 countries worldwide, coming together to raise awareness of this devastating rare disease, and to foster collaborations towards better medicines for patients.


For those who registered but couldn’t make it, or who couldn’t stay through the entire webinar, we have prepared a replay link. Please click HERE to watch the on-demand video.


At WuXi AppTec, we firmly believe in a future where “every drug can be made and every disease can be treated”. That future is only possible if we all work together.


Thank you again, and we hope to see you at our next episode of WuXi AppTec Rare Disease webinar series.

Agenda
  • 08:00 AM - 08:15 AM Welcome

    Hui Cai, VP and Head of Content, WuXi AppTec

    Pat Furlong, Founding President & CEO, Parent Project Muscular Dystrophy

    Debra Miller, CEO & Founder, CureDuchenne

    Filippo Buccella, Founder, Parent Project Italy APS

    Huigu Chen, President, Shanghai Jiai Myopathy Care Center, China

    Tali Kaplan, Co-Founder & CEO, Little Steps Association for Patients with Duchenne & Becker Muscular Dystrophy, Israel

    Sherena Loh, Director, Muscular Dystrophy Association (Singapore)

  • 08:15 AM - 08:45 AM Clinical Research and Practices

    Kevin Flanigan, Director, Center for Gene Therapy, Professor, Pediatrics & Neurology, Nationwide Children’s Hospital

    Stanley Nelson, Professor of Human Genetics, Center for Duchenne Muscular Dystrophy, David Geffen School of Medicine at UCLA

    Richard Parad, Associate Professor of Pediatrics, Harvard Medical School, Department of Pediatric Newborn Medicine, Brigham and Women’s Hospital

    Moderator: Hawken Miller, Features Writer, BioNews

  • 08:45 AM - 08:55 AM A Conversation

    A Conversation between Pat Furlong, Founding President & CEO, Parent Project Muscular Dystrophy and Debra Miller, CEO & Founder, CureDuchenne

  • 08:55 AM - 09:00 AM Patient Story - The Nilson Family
  • 09:00 AM - 09:30 AM What can we do to bring medicines to DMD patients around the world faster?

    Michael Binks, VP & Head of Clinical Research in the Rare Disease Research Unit at Pfizer

    Paula Clemens, Professor & Vice Chair, Neurology, University of Pittsburgh School of Medicine

    Nathalie Goemans, Professor, Neuromuscular Reference Centre for Children, Department of Pediatric Neurology, University Hospitals Leuven, Belgium

    Moderator: Timothy Franson, Principal, Faegre Drinker Consulting and formerly VP Global Regulatory Affairs, Eli Lilly & Co and Chief Medical Officer of YourEncore

  • 09:30 AM - 10:20 AM New Targets. New Modalities. New Directions.

    Rhonda Bassel-Duby, Professor, Department of Molecular Biology, University of Texas Southwestern Medical Center

    Ashish Dugar, SVP & Global Head of Medical Affairs, Dyne Therapeutics

    Jane Larkindale, VP, Clinical Sciences, PepGen

    Art Levin, CSO, Avidity Biosciences

    Stuart Peltz, Founder & CEO, PTC Therapeutics

    Moderator: Richard Soll, Head of Boston Office and Senior Advisor of Strategic Initiatives, WuXi AppTec

  • 10:20 AM - 10:30 AM 10-Year Vision

    Michael Binks, VP & Head of Clinical Research in the Rare Disease Research Unit at Pfizer

    Yi Dai, Associate Professor, Department of Neurology, Peking Union Medical College Hospital, China

    Xihua Li, Director, Department of Neuromuscular Disease, Children’s Hospital of Fudan University, China

    Stanley Nelson, Professor of Human Genetics, Center for Duchenne Muscular Dystrophy, David Geffen School of Medicine at UCLA

    Richard Parad, Associate Professor of Pediatrics, Harvard Medical School, Department of Pediatric Newborn Medicine, Brigham and Women’s Hospital

    Stacey Tay, Senior Consultant, Department of Paediatrics, Khoo Teck Puat - National University Children's Medical Institute, National University Hospital, Singapore

  • 10:30 AM - 10:35 AM Closing

    Hui Cai, VP and Head of Content, WuXi AppTec

Speakers
  • Hui Cai
    Hui Cai
    VP and Head of Content, WuXi AppTec
  • Pat Furlong
    Pat Furlong
    Founding President & CEO, Parent Project Muscular Dystrophy
  • Debra Miller
    Debra Miller
    CEO & Founder, CureDuchenne
  • Filippo Buccella
    Filippo Buccella
    Founder, Parent Project Italy APS
  • Huigu Chen
    Huigu Chen
    President, Shanghai Jiai Myopathy Care Center, China
  • Tali Kaplan
    Tali Kaplan
    Co-Founder & CEO, Little Steps Association for Patients with Duchenne & Becker Muscular Dystrophy, Israel
  • Sherena Loh
    Sherena Loh
    Director, Muscular Dystrophy Association (Singapore)
  • Kevin Flanigan
    Kevin Flanigan
    Director, Center for Gene Therapy, Professor, Pediatrics & Neurology, Nationwide Children’s Hospital
  • Stanley Nelson
    Stanley Nelson
    Professor of Human Genetics, Center for Duchenne Muscular Dystrophy, David Geffen School of Medicine at UCLA
  • Richard Parad
    Richard Parad
    Associate Professor of Pediatrics, Harvard Medical School, Department of Pediatric Newborn Medicine, Brigham and Women’s Hospital
  • Hawken Miller
    Hawken Miller
    Features Writer, BioNews
  • Michael Binks
    Michael Binks
    VP & Head of Clinical Research in the Rare Disease Research Unit at Pfizer
  • Paula Clemens
    Paula Clemens
    Professor & Vice Chair, Neurology, University of Pittsburgh School of Medicine
  • Nathalie Goemans
    Nathalie Goemans
    Professor, Neuromuscular Reference Centre for Children, Department of Pediatric Neurology, University Hospitals Leuven, Belgium
  • Timothy Franson
    Timothy Franson
    Principal, Faegre Drinker Consulting and formerly VP Global Regulatory Affairs, Eli Lilly & Co and Chief Medical Officer of YourEncore
  • Rhonda Bassel-Duby
    Rhonda Bassel-Duby
    Professor, Department of Molecular Biology, University of Texas Southwestern Medical Center
  • Ashish Dugar
    Ashish Dugar
    SVP & Global Head of Medical Affairs, Dyne Therapeutics
  • Jane Larkindale
    Jane Larkindale
    VP, Clinical Sciences, PepGen
  • Art Levin
    Art Levin
    CSO, Avidity Biosciences
  • Stuart Peltz
    Stuart Peltz
    Founder & CEO, PTC Therapeutics
  • Richard Soll
    Richard Soll
    Head of Boston Office and Senior Advisor of Strategic Initiatives, WuXi AppTec
  • Yi Dai
    Yi Dai
    Associate Professor, Department of Neurology, Peking Union Medical College Hospital, China
  • Xihua Li
    Xihua Li
    Director, Department of Neuromuscular Disease, Children’s Hospital of Fudan University, China
  • Stacey Tay
    Stacey Tay
    Senior Consultant, Department of Paediatrics, Khoo Teck Puat - National University Children's Medical Institute, National University Hospital, Singapore

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