• 10:00AM--Welcome
     Kathryn Bryant-Knudson, Founder of The Speak Foundation and Patient living with
    LGMD2i/R9
  • 10:05AM--FDA Speaker
    Wilson Bryan, MD, OTAT, CBER
  • 10:15AM--LGMD Clinical Overview
    Katherine Mathews, MD, University of Iowa
  • 10:30AM--Introduction & Meeting Overview
    James Valentine, JD, MHS - Co-moderator
    Jennifer Levy, PhD, Coalition to Cure Calpain 3 - Co-moderator
  • 10:35AM--Demographic polling
    Includes all 6 LGMD subtypes
  • 10:40AM--Session 1: Living with Sarcoglycanopathies (LGMD2C, 2D, 2E, 2F) – symptoms, health effects, and impacts on daily life
    ● Includes Patient Panelists and Discussion Starters
    ● Patient/caregiver audience remote polling
    ● Moderated audience discussion (telephone and written comments)
  • 11:40AM--Session 2: Living with LGMD2A and LGMD2I – symptoms, health effects, and impacts on daily life
    ● Includes Patient Panelists and Discussion Starters
    ● Patient/caregiver audience remote polling
    ● Moderated audience discussion (telephone and written comments)
  • 12:40PM--Lunch
  • 1:20PM--Session 3: Current and Future Treatments (all 6 subtypes)
    ● Includes Patient Panelists and Discussion Starters
    ● Patient/caregiver audience remote polling
    ● Moderated audience discussion (telephone and written comments)
  • 2:45PM--Summary Remarks
    Larry Bauer, RN, MA
  • 2:50PM--Closing Remarks & Next Steps

    Charlotte Drew, MD - Co-Founder of The Kurt+Peter Foundation and Caregiver
    of two sons living with LGMD2C/R5
  • 3:00PM--Adjourn

Externally-Led Patient Focused Drug Development Meeting for Limb-Girdle Muscular Dystrophies Types 2A, 2I,  and Sarcoglycanopathies


September 23, 2022

ALL TIMES ARE IN EASTERN DAYLIGHT TIME (EDT); UTC -4