Limb-Girdle Muscular Dystrophy Scientific Leaders – Together With the FDA – Will Come Together for a Drug Development Workshop on February 8, 2024

ROCKVILLE, Md., Dec. 13, 2023 /PRNewswire/ — On February 8, 2024, The Speak Foundation, a patient-led 501(c)(3) organization for Limb-Girdle Muscular Dystrophy (LGMD), will be convening a multi-stakeholder LGMD Scientific Drug Development Workshop in Rockville, Maryland. The workshop will continue to drive the momentum for the sense of urgency needed by the patient community to accelerate LGMD drug development.

Academic medical experts, senior leaders from the U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER), highly experienced drug developers, and other specialists will come together to discuss LGMD pathophysiology, clinical outcomes and patient-focused drug development, surrogate endpoints and the accelerated approval pathway. The meeting will focus on six LGMD subtypes: LGMD2A/R1, LGMD2B/R2, LGMD2C/R3, LGMD2D/R4, LGMD2E/R5, and LGMD2I/R9.

“In September 2022, at the LGMD community’s first Externally-Led Patient Focused Drug Development Meeting (EL-PFDD), the FDA heard perspectives of patients and caregivers on the health effects, daily impacts, treatment goals, and decision factors that would be considered when seeking out or selecting a treatment,” explains Kathryn Bryant, founder of the Speak Foundation. “This workshop is a continuing step to work with the FDA and LGMD scientific leaders on pushing critical drug development forward. The time is now.”

LGMD Scientific Workshop session presenters will include CBER Director Dr. Peter Marks, and CDER’s Office of New Drugs (OND) Director, Dr. Peter Stein. Other confirmed speakers include Dr. Jerry R. Mendell, retired attending neurologist of Nationwide Children’s Hospital, Dr. Nicholas Johnson of Virginia Commonwealth University, Dr. Louise Rodino-Klapac of Sarepta Therapeutics, Dr. Douglas Sproule of ML Bio Solutions, a BridgeBio affiliate, Dr. Katherine Mathews of University of Iowa Health Care, Dr. Matthew Wicklund of University of Texas San Antonio, Dr. Peter Kang of University of Minnesota Medical School and Dr. Lindsay Alfano of Nationwide Children’s Hospital. 

“As a translational clinician scientist, devoting a lifetime of work toward improving the lives of patients with muscular dystrophy, workshops like the LGMD Scientific meeting are important venues for discussing the current scientific understanding of LGMD and the implications for drug development.” said Dr. Jerry R. Mendell. “Bringing key LGMD stakeholders together to share ideas and research findings in a public forum is the most productive way to find solutions and advance therapies that will improve the lives of LGMD patients.”

The LGMD Scientific Workshop will be a focused discussion with clinicians, scientific researchers, drug developers, regulators, patients and patient advocates on the characteristics of LGMD that drive drug development programs. The meeting will be held virtually and by invitation for in-person attendance. Proceedings will be recorded and shared with the broader patient and scientific communities through The Speak Foundation. More details will be added soon to: The Speak Foundation – Limb Girdle Muscular Dystrophy.

About The Speak Foundation:

The Speak Foundation is a 501(c)(3) nonprofit charity, which is focused on supporting patients living with all forms of LGMD and on facilitating development of treatments for these diseases.

About Limb Girdle Muscular Dystrophy:

Limb-Girdle Muscular Dystrophy (LGMD) is a group of muscular dystrophies caused by mutations in one of over 30 genes. The age of onset, symptoms, progression, and inheritance pattern varies among genetic subtypes, but generally result in severe disability and are often life-threatening due to cardiac and respiratory complications. There are estimated to be some 20,000+ people living with a form LGMD in the United States.

Media Contact:
Rachel DeConti


SOURCE The Speak Foundation