On September 23, 2022, a coalition of limb-girdle muscular dystrophy (LGMD) organizations held an Externally-Led Patient Focused Drug Development (EL-PFDD) meeting for six LGMD subtypes: 2C (R5), 2D (R3), 2E (R4), 2F (R6), 2A (R1), and 2i (R9). The LGMD Coalition includes the following organizations: Coalition to Cure Calpain 3, CureLGMD2i, Kurt+Peter Foundation, LGMD2D Foundation, McColl-Lockwood Laboratory for Muscular Dystrophy Research, and the Speak Foundation.
The goal of this meeting was to provide the U.S. Food and Drug Administration (FDA), product developers, clinicians, industry, and academic researchers an opportunity to hear perspectives from individuals with LGMD on the health effects, daily impacts, treatment goals, and decision factors considered when seeking out or selecting a treatment. This meeting was conducted as a parallel effort to FDA’s EL-PFDD initiative, a commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) to more systematically gather patients’ perspectives on their conditions and available therapies to treat their conditions.
Please note that this initial endeavor represented Phase 1 and included six LGMD subtypes. We anticipate additional LGMD subtypes will pursue EL-PFDD meetings in the future.
Below on this page you can view the recorded footage of the meeting or read the summary of the meeting in our Voice of the Patient (VOP) report. You can also find more information about this EL-PFDD meeting at this link:
Watch the first ever Limb-Girdle Muscular Dystrophy Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting held on September 23, 2023.
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