FALLS CHURCH, Va., July 5, 2022 /PRNewswire/ — On September 23, 2022 at 10am EDT, a coalition of limb-girdle muscular dystrophy (LGMD) nonprofit organizations will lead an Externally-Led Patient Focused Drug Development Meeting (EL-PFDD). This online event is open to the public with registration required. The meeting will be summarized in a Voice of the Patient report which, along with recorded footage of the meeting, will be publicly available.
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 meeting is being conducted as a parallel effort to FDA’s EL-PFDD initiative, a commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) to more systematically gather patients’ perspectives on their conditions and available therapies to treat their conditions.
The meeting agenda will include speakers and panelists who are living with LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2I/R9, as well as caregivers of people living with these six subtypes. The morning sessions will focus on symptoms that matter most to them, and the toll these take on daily living. The afternoon session will include discussions on experiences with current treatments and attitudes towards future treatments, including tolerance to potential risks.
For the first time, representatives from 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 joined together to organize this event. Jointly referred to as the LGMD Coalition, these nonprofit organizations are encouraging members of the global patient community to make their voices heard. 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. Pre-registration is available at https://LGMDPFDD.com.
The LGMD Coalition is grateful for support from our sponsors, AskBio, Edgewise Therapeutics, ML BioSolutions, Sarepta Therapeutics, and Vita Therapeutics. We also acknowledge our Advocacy Partners, the Muscular Dystrophy Association and the LGMD Awareness Foundation.
LGMD is a term used for a group of rare neuromuscular diseases which are inherited and cause muscle weakness and wasting. The muscles most affected are those closest to the body (proximal), specifically the muscles of the shoulders, upper arms, pelvic area, and thighs. More than 30 subtypes of LGMD exist. Together, LGMDs have an estimated prevalence of about 2 in every 100,000 individuals. There is currently no cure for any subtype of LGMD.
The LGMD Coalition is a group of six 501(c)3 nonprofit 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 – that are collaborating to host an LGMD EL-PFDD focused on subtypes LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2I/R9. Learn more at https://LGMDPFDD.com.
SOURCE The LGMD Coalition