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 EL-PFDD meeting was designed to engage patients and elicit their unique perspectives on the health effects, daily impacts, treatment goals, and decision factors considered when seeking out or selecting a treatment for symptoms and burdens associated with LGMD subtypes 2C, 2D, 2E, 2F (collectively termed sarcoglycanopathies), LGMD2A, and LGMD2i. The meeting was held virtually to enable as many community members to participate as possible and to allow many different voices to be heard. The meeting was conducted as a parallel effort to FDA’s 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.

Click on the image below to download a PDF copy of the Voice of the Patient Report, which provides a detailed summary of the outcomes of the EL-PFDD meeting. Select patient comments collected in a pre-meeting survey and through an online portal are available in a separate PDF document.