Scientific Updates on Organic Acidemias and Homocystinurias - Panel Discussion (Day 2)

The National Human Genome Research Institute (NHGRI) and the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health co-hosted a virtual meeting on “Scientific Updates on Organic Acidemias and Homocystinurias.” This meeting brought 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. Opportunities and challenges that patients and their physicians face in several upcoming small molecule, enzyme replacement and genomic phase I/II clinical trials were also discussed.