September Online Meeting to Highlight Views of LGMD Patients
Coalition will host externally-led patient-focused drug development meeting
by |
A coalition of six non-profits will host a patient-focused meeting in September for six forms of limb-girdle muscular dystrophy (LGMD).
The Sept. 23 online event is free, open to the public, and will focus on LGMD 2A (R1), 2C (R5), 2D (R3), 2E (R4), 2F (R6), and 2I (R9). Participants will need to pre-register to attend.
LGMD is a group of rare disorders that causes muscle wasting at the hip and shoulders. There are over 30 forms of LGMD, and although muscle weakness is common among them, the age of onset, inheritance patterns, and disease progression are different between subtypes.
This is the first time the six 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 — have come together to organize this event, called an externally-led patient-focused drug development meeting, according to a press release.
Overall, the meeting seeks to provide the U.S. Food and Drug Administration, clinicians, researchers, and medical product developers with perspectives from patients, caregivers, and families on the impact of LGMD, treatment goals, and factors when considering therapies. The information can influence regulatory decisions and oversights during treatment development and review of marketing applications for new therapies.
The meeting’s agenda includes morning discussions that will focus on disease symptoms and their effects on daily life, and afternoon sessions about available and potential treatments from the patient perspective.
Live speakers, pre-recorded panelists, and live audience polling and discussion forums are also planned for the day. Patients and their caregivers are encouraged to participate by submitting comments online before the meeting, and by answering polling questions, submitting written comments, and calling in on the day of the meeting.
The information shared during the meeting will be summarized in a Voice of the Patient Report that will be publicly available as an educational reference document and include recorded footage.
Current supporters of the LGMD coalition include Sarepta Therapeutics, AskBio, Edgewise Therapeutics, ML BioSolutions, and Vita Therapeutics.
About the Author
