Achondroplasia
Your Guide to Assessment, Management, and Coordination of Care
Monday, October 5, 2020 | 7:00 PM - 8:30 PM CDT
    LIVE Virtual Symposium

    Participants can access this live virtual event on Monday, October 5, from 7:00 – 8:30 PM CDT.

    Activity Overview

    Achondroplasia (Ach), the most common form of short limb dwarfism in humans, affects 250,000 people worldwide. Although short stature with rhizomelic limb-shortening and macrocephaly are hallmark features of Ach, there are a number of medical complications associated with Ach that significantly increase morbidity and mortality in these patients. Treatment of the developmental complications of Ach currently involves symptomatic management, surgical intervention, and lifelong follow-up care. New therapeutic approaches targeting elements in molecular disease mechanisms of Ach are now in development. These novel investigative agents aim to restore bone growth and prevent many of the medical conditions associated with Ach.

    In this virtual live symposium, an expert panel will address the burden of disease and complications associated with Ach, benefits and limitations of current care recommendations, emerging therapies for Ach, and strategies for coordination of care with specialists.

    Speakers
    • Carlos A. Bacino, MD
      Moderator
      Professor of Molecular and Human Genetics
      Vice Chair of Clinical Affairs
      Department of Molecular and Human Genetics
      Baylor College of Medicine
      Director
      Pediatric Clinical Genetics Service
      Texas Children's Hospital
      Houston, Texas
    • Michael B. Bober, MD, PhD
      Panelist
      Director
      Skeletal Dysplasia Program
      Nemours/Alfred I. duPont Hospital for Children
      Professor of Pediatrics
      Thomas Jefferson University’s Stanley Kimmel Medical College
      Wilmington, Delaware
    • Julie Hoover-Fong, MD, PhD
      Panelist
      Professor McKusick-Nathans Department of Genetic Medicine
      Director
      Greenberg Center for Skeletal Dysplasias
      Johns Hopkins University School of Medicine
      Baltimore, Maryland
      Participation by Dr Julie Hoover-Fong does not constitute or imply endorsement by the Johns Hopkins University or the Johns Hopkins Hospital and Health System.
    • William R. Wilcox, MD, PhD
      Professor of Human Genetics
      Emory University School of Medicine
      Atlanta, Georgia
      The opinions expressed are those of Dr William R. Wilcox and do not necessarily reflect the views of Emory University or Emory Healthcare. Dr William R. Wilcox’s participation in this activity does not constitute or imply endorsement by Emory University or Emory Healthcare.
    Program Monday, October 5, 2020
    • 7:00 PM
    • Introduction
    • 7:10 PM
    • Case Presentations
      • Burden of Disease and Complications in Achondroplasia
      • Current Treatments and Unmet Clinical Needs
      • Coordination of Care With the Multidisciplinary Team
    • 8:05 PM
    • Emerging Therapies in Achondroplasia
    • 8:15 PM
    • Concluding Remarks
    • 8:20 PM
    • Q&A
    Learning Objectives
    • Have increased knowledge regarding the
      • Burden of disease in individuals with Ach
      • Limitations of current care recommendations for Ach
      • Emerging therapies for Ach
    Target Audience
    Goal Statement
    Association Disclaimer Statement
    Credits Available
    • 1.5
    • For Physicians
    Accreditation Statement
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    Supported by an independent educational grant from
    BioMarin