New 'Voice of the Patient' Report Highlights Unmet Medical Needs of Six Subtypes of Limb-Girdle Muscular Dystrophy
This meeting focused on LGMD subtypes LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2i/R9.
- This meeting focused on LGMD subtypes LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2i/R9.
- An additional 136 patients and family members responded to a live survey and over 250 written comments were submitted.
- The LGMD Coalition is appreciative for support from our sponsors, Sarepta Therapeutics, AskBio, Edgewise Therapeutics, ML BioSolutions, and Vita Therapeutics.
- We believe that their engagement in this process provided insights into these subtypes of LGMD which will help the research community develop effective therapies."