New “Voice of the Patient” report highlights unmet medical needs of six subtypes of LGMD, including LGMD2A/R1

Today, Coalition to Cure Calpain 3 joins five other LGMD patient organizations (collectively referred to as The LGMD Coalition*) to release the ‘Voice of the Patient Report’ summarizing outcomes from the September 2022 Externally-Led Patient Focused Drug Development Meeting (EL-PFDD).

The goal of the report is to provide the U.S. Food and Drug Administration (FDA), clinicians, medical product developers, 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. The meeting was conducted as a parallel effort to FDA’s EL-PFDD initiative to more systematically gather patients’ perspectives on their conditions and available therapies to treat their conditions.

Click here to visit LGMDPFDD.com and access the report and video recording of the September 23, 2022 meeting.

* ABOUT THE LGMD COALITION

The LGMD Coalition is a group of six 501(c)3 nonprofit organizations — Coalition to Cure Calpain 3, CureLGMD2I, the Kurt+Peter Foundation, the LGMD2D Foundation, the McColl-Lockwood Laboratory for Muscular Dystrophy Research, and the Speak Foundation – that collaborated to host an LGMD EL-PFDD focused on subtypes LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2I/R9. The LGMD Coalition is appreciative for support from our sponsors: Sarepta Therapeutics, AskBio, Edgewise Therapeutics, ML BioSolutions, and Vita Therapeutics.