LANCASTER, Pa., Jan. 20, 2025 -- On July 18, 2025, The Speak Foundation will host the fourth International Limb Girdle Muscular Dystrophy (LGMD) Conference at the J.W. Marriott Orlando, Grande Lakes.
The International LGMD conference brings together patients, caregivers, physicians, researchers, and advocacy groups representing all forms of LGMD. Leading experts in the field will share cutting-edge research and drug development updates across multiple forms of the LGMDs. LGMD patients and families are encouraged to attend to learn, network and enjoy all that the Orlando area has to offer.
Geared largely towards the patient and advocacy community, the International LGMD Conference provides the latest updates on diagnostic and medical management, natural history studies, preclinical research of new potential treatment modalities, clinical trials and development of treatments.
There will be a number of "breakout sessions" at the Conference focused on building community connections, educating on opportunities to advocate for LGMD, and providing updates on strategies for independent living. These will include both tips from clinicians on different aspects of disease management and peer-to-peer advice on living one's best life with LGMD. This conference presents a great opportunity to meet and connect with others in the LGMD community.
At the conference two experts in the field will be honored with awards for Lifetime Achievement and Clinical Excellence in the LGMDs. Confirmed speakers include Dr. Volker Straub of the University of Newcastle, Dr. Louise Rodino-Klapac of Sarepta Therapeutics, Dr. Sharon Hesterlee of MDA, Dr. Douglas Sproule of ML Bio Solutions, Dr. Matthew Wicklund of University of Texas San Antonio, Dr. Conrad (Chris) Weihl of Washington University School of Medicine, Dr. Peter Kang of University of Minnesota, Dr. Kevin Campbell of University of Iowa and Dr. Barry Byrne of University of Florida.
"We are proud to stand alongside this remarkable community," said Douglas Sproule, M.D., M.Sc., Chief Medical Officer of ML Bio Solutions, a BridgeBio company developing BBP-418 for LGMD2I/R9, "and contribute to a gathering that empowers people living with all forms of LGMDs, fosters connection, and advances knowledge. Together, we can make a difference."
"Witnessing the remarkable growth of the LGMD community with each conference has been truly inspiring. As advancements in LGMD treatment and care continue, the connections we've built and our collective readiness to learn and adapt will play crucial roles in driving change. A heartfelt thank you to the Speak Foundation and every organization that has contributed to the growth of this community and, more specifically, to the success of this conference," said Louise Rodino-Klapac, PhD, Executive Vice President and Chief Scientific Officer, Sarepta Therapeutics.
The Speak Foundation gratefully acknowledges the generous support from the conference's platinum level sponsors. Sarepta Therapeutics is developing gene therapy treatments for several genetic subtypes of LGMD with important ongoing natural history studies, pre-clinical, and clinical programming underway. ML Bio Solutions is developing a small molecule therapy targeted to LGMD 2I/R9, which is caused by mutations in the FKRP gene.
Registration is now open. Visit www.internationallgmdconference.com to register and book your hotel stay as space is limited.
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.
About ML Bio Solutions
ML Bio Solutions (ML Bio), a BridgeBio company, is a biotechnology company founded in 2018, yet the company's founders have been dedicated to finding a cure for Limb-Girdle Muscular Dystrophy Type 2I/R9 FKRP-related (LGMD R9/2I) for close to twenty years. ML Bio is developing BBP-418, potentially the first oral treatment for patients with LGMD 2I/R9. www.mlbiosolutions.com
About Sarepta Therapeutics
Sarepta Therapeutics is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and Limb-Girdle Muscular Dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. https://www.sarepta.com
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