Limb-girdle muscular dystrophy coalition to host externally-led patient focused drug development meeting

C3 is excited to announce that we’ve teamed up with the LGMD2D Foundation, the Kurt+Peter Foundation, CureLGMD2I, the McColl-Lockwood Laboratory for Muscular Dystrophy Research, and the Speak Foundation to host an Externally-Led Patient Focused Drug Development (EL-PFDD) meeting with the U.S. Food and Drug Administration and other key stakeholders.

This EL-PFDD will focus on six subtypes of LGMD: LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2I/R9. The goal of this meeting 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.

This online event is open to the public with registration required. Individuals living with the subtypes of LGMD listed above, as well as their caregivers, are invited to participate in the meeting by partaking in live polling and by calling and writing in with comments. Academic investigators, clinicians, regulators, and industry representatives are encouraged to watch the meeting and incorporate patient/caregiver perspectives when developing and reviewing new therapies to best meet the needs and expectations of our community.

Preregistration is available at https://LGMDPFDD.com. The meeting will be summarized in a Voice of the Patient report which, along with recorded footage of the meeting, will be publicly available.