LANCASTER, Pa. — On October 27, 2023, the third International Limb Girdle Muscular Dystrophy (LGMD) Conference will be hosted by The Speak Foundation at the Grand Hyatt Washington, DC.
The inaugural International LGMD conference was held in Chicago in 2019 and over 400 patients, caregivers, physicians, researchers and advocacy groups representing ALL forms of LGMD came together to inspire hope for our LGMD community. The second International LGMD conference was in 2021 and was offered in a virtual format due to COVID-19. The conference will be back in person this year and will be offering live-streamed sessions for those unable to attend. The leading experts in the field will be sharing cutting edge research and drug development updates across multiple forms of LGMDs.
The keynote speaker is Dr. Peter Marks, head of the Center for Biologics Evaluation and Research (CBER) at the FDA. Dr. Marks will discuss the challenges in developing therapies for ultra-rare diseases and some of the initiatives that FDA and its partners have undertaken to facilitate therapy development. Other confirmed speakers for the Conference include Dr. Carsten Bonnemann of the NIH, Dr. Volker Straub of the University of Newcastle, Dr. Louise Rodino-Klapac of Sarepta Therapeutics, Dr. John Vissing of the University of Copenhagen, Dr. Sharon Hesterlee of the MDA, Dr. Nicholas Johnson of Virginia Commonwealth University, Dr. Douglas Sproule of ML Bio Solutions, and Dr. Linda Lowes of Nationwide Children’s Hospital.
The LGMD Conference is geared largely towards the patient and advocacy community, informing them of the latest updates on diagnosis, clinical trial readiness, and development of treatments. Sessions will cover natural history studies, diagnosis, preclinical development of new treatment modalities, and updates on clinical trials. Taking advantage of this year’s location in Washington D.C., there will be a special section on advocacy initiatives for LGMDs.
There will be a number of “breakout sessions” at the Conference geared toward providing practical advice to the patient community. These will include both tips from clinicians on different aspects of disease management, 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.
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: R1/2A, R2/2B, R3/2D, R4/2E, R5/2C, and R12/2L. ML Bio Solutions is developing a small molecule therapy targeted to LGMD R9/2I, which is caused by mutations in the FKRP gene.
Registration opens April 5. Visit www.internationallgmdconference.com to register.
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 R9/2I. 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