FALLS CHURCH, Va., July 5, 2022 /PRNewswire/ — About September 23, 2022 at 10 a.m. EDT, a coalition of nonprofit organizations specializing in limb-girdle muscular dystrophy (LGMD), will lead an external meeting on patient-oriented drug development (EL-PFDD). This online event is open to the public with mandatory registration. The meeting will be summarized in a Patient’s Voice report which, along with recorded footage of the meeting, will be available to the public.
This EL-PFDD will focus on six LGMD subtypes: LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2I/R9. The purpose of this meeting is to provide the U.S. Food and Drug Administration (FDA), clinicians, medical product developers, and academic researchers with an opportunity to hear the perspectives of people with LGMD. on health effects, daily impacts, treatment goals and decision factors. considered when researching or selecting a treatment. This meeting is being conducted in conjunction with the FDA’s EL-PFDD initiative, a commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) to more systematically gather patient perspectives on their conditions and therapies available to treat their conditions.
The meeting agenda will include speakers and panelists who live 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. Morning sessions will focus on the symptoms that matter most to them and how they impact daily life. The afternoon session will include discussions on experiences with current treatments and attitudes towards future treatments, including tolerance for potential risks.
For the first time, representatives from the 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. Collectively known as the LGMD Coalition, these nonprofit organizations encourage members of the global patient community to raise their voices. People living with the LGMD subtypes listed above, as well as their caregivers, are invited to participate in the meeting by participating in a live survey and by calling and writing with comments. Academic researchers, clinicians, regulators and industry representatives are encouraged to watch the meeting and incorporate patient/caregiver perspectives when developing and reviewing new therapies to better meet the needs and expectations of our community. Pre-registration is available at https://LGMDPFDD.com.
The LGMD Coalition is grateful for the support of our sponsors, AskBio, Edgewise Therapeutics, ML BioSolutions, Sarepta Therapeutics and Vita Therapeutics. We also thank our advocacy partners, the Muscular Dystrophy Association and the LGMD Awareness Foundation.
LGMD is a term used for a group of rare neuromuscular diseases that are inherited and cause muscle weakness and wasting. The muscles most affected are those closest to the body (proximal), especially the muscles of the shoulders, arms, pelvic region and thighs. More than 30 subtypes of LGMD exist. Together, LGMDs have an estimated prevalence of about 2 in 100,000 people. There is currently no cure for any subtype of LGMD.
About the LG® Coalition
The LGMD Coalition is a group of six 501(c)3 nonprofit organizations — Coalition to Cure Calpain 3, CureLGMD2I, Kurt+Peter Foundation, LGMD2D Foundation, McColl-Lockwood Laboratory for Muscular Dystrophy Research, and Speak Foundation – who are collaborating to host an LGMD EL-PFDD focused on LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2I/R9 subtypes. Learn more at https://LGMDPFDD.com.
SOURCE The LG® Coalition