On October 20th, 2020, the LGMD community was able to meet with the FDA to share our patient experiences. This was a patient-led listening session organized by a consortium of advocacy organizations, including Coalition to Cure Calpain 3. The session focused on various aspects and genetic subtypes of LGMD. Fifteen presenters, all patients or family members representing twelve LGMD subtypes, gave presentations in a webinar format which included personal narratives, slide presentations, and videos.
The FDA created the Rare Disease Patient Listening Sessions to offer patients and caregivers an opportunity to speak directly to FDA staff about what it’s like to live with a specific rare disease. They helps FDA staff understand the patients’ perspective about their disease, needed treatment options, and meaningful outcomes. Participants provide unique perspectives about the reality of living with a rare disease or caring for someone who does.