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The National Human Genome Research Institute (NHGRI) and the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health will co-host a virtual meeting on “Scientific Updates on Organic Acidemias and Homocystinurias.”
For over 15 years, the Organic Acid Research Section (OARS) of NHGRI has worked on organic acidemias with clinical and scientific partners to develop genomic and small molecule therapies for a number of the enzymatic defects in the propionyl-CoA and vitamin B12 (cobalamin) metabolic pathways.
This meeting will bring together researchers, clinicians, families and patient advocates from around the world to discuss the natural history, disease pathophysiology, genetics and treatments for rare inborn metabolic errors. We will also review the opportunities and challenges that patients and their physicians face in several upcoming small molecule, enzyme replacement and genomic phase I/II clinical trials.
We hope that you will join us for an exciting two days on this fascinating topic.