Upcoming LGMD Events

On February 8, 2024, The Speak Foundation convened several academic medical experts, senior leaders from the U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER), highly experienced drug developers, and other specialists that all sought to answer important questions on how to drive momentum to fulfill the unmet, yet critical need of the patient community to accelerate drug development for LGMDs. The meeting focused on six LGMD subtypes: 2A/R1, 2B/R2, 2C/R5, 2D/R3, 2E/R4, and 2I/R9.    

The LGMD Scientific Workshop provided scientific and medical experts, regulators, patients, and drug development stakeholders the opportunity to explore innovative drug development approaches and clinical trial designs targeting the causal pathways and appropriate clinical and surrogate endpoints tailored for each subtype.  

  The first externally led patient-focused drug development (EL-PFDD) meeting for LGMD was held on September 23,2022. This monumental event gave individuals diagnosed with limb-girdle muscular dystrophy types 2A, 2C, 2D, 2E, 2F, and 2I an opportunity to share with the FDA and other stakeholders about the experiences and challenges of living with LGMD.