sEPTEMBER 23, 2022

The LGMD Coalition:
The LGMD community will be spearheading an Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting on Friday, September 23, 2022, for six limb-girdle muscular dystrophy (LGMD) subtypes: 2C (R5), 2D (R3), 2E (R4), 2F (R6) 2A (R1), and 2I (R9).
What is an externally-led patient-focused drug development meeting for LGMD?
EL-PFDD meetings give the Food and Drug Administration (FDA) and other key stakeholders — including medical product developers, health care providers, and federal partners — an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight both during drug development and during their review of a marketing application for a new drug.

This free public meeting will be virtual.  The interactive meeting will include live speakers, pre-recorded panelists, and – throughout the day – live polling and discussion forums featuring YOU, our viewing audience. The morning session will focus on sub-type symptoms, their health effects, and the toll these six forms of LGMD take on your daily living. The afternoon session will highlight our community’s perspectives on currently available and future treatments and communicate our tolerance to risk. See the EL-PFDD Agenda
How can I participate?
This interactive meeting is designed to engage patients living with LGMD 2A (R1), 2C (R5), 2D (R3), 2E (R4), 2F (R6), and 2i (R9), and their caregivers and families, to elicit their unique perspectives. There are three ways you can participate.
1. If you or a loved one is living with one of these subtypes of LGMD, please submit pre-meeting comments online so that they may be included in the meeting or the Voice of the Patient report.
Submit Pre-Meeting Comments
2. You will also be able to participate on the day of the EL-PFDD meeting by answering polling questions, submitting written comments, and calling in. To attend this interactive virtual meeting please pre-register through our Eventbrite site. This is a FREE event.
3. On September 23, 2022, access the interactive EL-PFDD meeting through this website. Your voice will make a difference! We look forward to your participation.
What happens after the meeting?
The ideas and information shared during the meeting will be consolidated into a
summary meeting report known as a Voice of the Patient Report. The publicly available report will serve as an educational reference document for the FDA, medical product developers, health care providers, federal partners, and the LGMD community. The Voice of the Patient report will be available to the public on this website as well as have a link on the FDA website.

To learn more about EL-PFDDs, please check out the
FDA's EL-PFDD web page.
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