Thank you to our Platinum Sponsors!
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 (LGMD2I/LGMDR9) for close to twenty years. ML Bio is developing BBP-418, potentially the first oral treatment for patients with LGMD2I.
Sarepta 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.
Thank you to our Bronze Sponsors!
Vita Therapeutics is a cell engineering company that aims to develop life translational treatments. It was founded out of Johns Hopkins University in 2019 by Douglas Falk, M.S. and Peter Andersen, PhD. The company utilizes induced pluripotent stem cell (iPSC) technology to engineer specific cell types designed to replace those that are defective in patients. Our first product is called VTA-100 and is an autologous stem cell product aimed at treating individuals with Limb-Girdle Muscular Dystrophy Type 2A. The product starts with a venous blood draw from an affected individual. The iPSCs are generated from this sample and then a corrected copy of Calpain-3 is inserted into the stem cells. These are differentiated into to satellite cells, the stem cells of skeletal muscles, and then reintroduced into the patient’s affected muscles to recover biological function, support true regeneration, and cure disease at the root.
AskBio, an independent subsidiary of Bayer AG, is an integrated gene therapy company dedicated to improving patient outcomes with transformative genetic medicines and scalable manufacturing. Its pipeline includes therapeutics for neuromuscular, central nervous, metabolic, and cardiovascular conditions. Global headquarters are in Research Triangle Park, NC, and European headquarters in Edinburgh, UK.
Thank you to our Partners of Strength!
At Atamyo, we work on the development of novel approaches to treat different forms of limb-girdle muscular dystrophies (LGMD), gene-related disabling diseases characterized by a predominant impairment of muscles of the hip and shoulder areas. We use our expertise in gene therapy and muscular dystrophies to find new approaches for gene replacement. Our goal is to design the best delivery system for a particular disorder, in order to decrease side effects and improve the restoration of a normal physiological function for patients. Atamyo’s therapeutic strategies are based on modified adeno-associated virus (AAV). In collaboration with Genethon's research teams, we design new capsids, promoters, transgenes and regulation elements that will increase power, specificity and safety of future treatments.
Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on creating novel precision medicines for the treatment of rare muscle disorders. Our intimate knowledge of integrated muscle physiology at a whole-body level allows us to develop innovative solutions for patients with muscle disease where significant unmet medical need exists. By protecting and improving muscle health, our goal is to dramatically enhance the lives of people living with progressive muscle disorders.
Thank you to our LGMD organization Sponsors!
The CureLGMD2i Foundation, a 501(c)3 non-profit organization, was founded in 2010 by the Brazzo family. Their mission was to provide funding toward the development of a safe and effective treatment for Limb Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9). CureLGMD2i is focused on providing advocacy, spreading awareness and supporting scientific research toward a cure for LGMD2I/R9.
The LGMD2D Foundation is a non-profit organization built for families living with Limb-Girdle Muscular Dystrophy, type 2D / R3 - both patients and caregivers - by families with the same diagnosis. Our mission is to support research and clinical trials to speed the development of treatments and a cure for LGMD2D.
The LGMD2L Foundation’s goal is to unite people afflicted by the rare disease Limb-girdle muscular dystrophy type 2L (LGMD2L). Also, provide an open forum to connect, discuss, and ultimately build a community of LGMD2L members. Although our disease is rare and poorly understood, we have established this foundation to build a database of LGMD2L patients for future scientific research and clinical trials. The only way to bring about change is to unify those who are driven for a cure!
LGMD Awareness Foundation, Inc. is a 501(c)(3) advocacy organization dedicated to globally raising awareness of the rare neuromuscular diseases known as limb-girdle muscular dystrophy (LGMD). Our focus is to provide curated educational information and resources for the LGMD community, inclusive of all LGMD subtypes and those without a subtype-specific diagnosis. We are a patient-led organization, as all members of our Board of Directors live with LGMD. Our Advisory Board consists of representatives from many LGMD organizations. In collaboration with other LGMD foundations, we also coordinate LGMD Awareness Day which is celebrated worldwide on September 30th each year.
The LGMD-1D DNAJB6 Foundation is a non-profit public foundation whose mission is to maintain a central location for individuals diagnosed with Limb Girdle Muscular Dystrophy type 1D (LGMD1D) to meet, consolodate relevent news, and launch fundraising efforts in order to advance research. Genetic and molecular research for these orphan diseases are exploding and techniques that are developed to cure can often be shared with others often afflicted with other crippling genetic disorders that are seemingly unrelated