Development of Targeted Therapies for Peroxisome Biogenesis Disorders: Current and future prospects

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The Development of Targeted Therapies for Peroxisome Biogenesis Disorders: Current and future prospects conference will provide a forum for multidisciplinary investigators to discuss emerging therapeutic opportunities for peroxisome biogenesis disorders (PBDs). Our overarching goal is to develop a roadmap for PBD therapeutic development based on the discussions that originate from and extend beyond this conference. PBDs are a disease spectrum that impacts the functions of multiple organ systems, with the most profound effects on the central nervous system, and leads to a shortened life span. In addition to the neurological aspects of disease, patients most typically manifest a progressive loss of vision and hearing as well as respiratory, hepatic, renal, and skeletal abnormalities. The therapeutic roadmap generated as a result of this conference will be used by clinically-oriented colleagues and relevant disease foundations, the Global Foundation for Peroxisomal Disorders (GFPD) and RhizoKids International, to plan for future translational research initiatives. The audience will be comprised of a diverse community of individuals with complementary areas of expertise including physician-scientists who treat patients with PBDs, translational researchers, basic scientists, and patient advocates, all of whom share an interest in therapeutic initiatives for rare diseases, including PBDs. Special emphasis will be placed on the participation of junior investigators and trainees, with the expectation that they will provide novel ideas and perspectives and also benefit from interacting with the community-based therapeutic planning efforts at the conference. Participants from underrepresented communities in the neurosciences and general medical sciences will provide important perspectives and be involved in critical discussions about patient recruitment and access to newly developed treatments. The diverse scientific community and patient advocates participating at this conference will allow for a cross-fertilization of ideas relevant to translational research that may not be achievable in more general meetings, such as those of the American Society of Gene and Cell Therapy (ASGCT) and the American Society of Human Genetics (ASHG).