Cure RBM is supporting the work of the world’s leading researchers in gene therapy and Reducing Body Myopathy. We are very optimistic about the potential for life-saving gene therapies involving adeno-associated viruses (AAVs). The malfunctioning of the mutated FHL1 gene is what causes Reducing Body Myopathy. The goal of a disease-halting gene therapy is to knock down the mutated gene and replace it with a healthy functioning copy. One of the critical considerations of developing a treatment is to be able to address dominant negative component in this disease, and several strategies will need to be evaluated to arrive at the best candidate vector. With your help, the foundation is committed to providing funding to continue the research and development of this potentially lifesaving therapy.