Rare Disease Week, the FDA, mortality in autism, trajectories and subcategories
About this episode
This episode discusses highlights in scientific research related to rare diseases, FDA actions, mortality in autism, and genetic predictions.
This week’s podcast summarizes some highlights in scientific research and includes a recognition of Rare Disease Week and actions taken by the FDA to ease the criteria for evaluating genetic therapies for rare genetic disorders, the best study to date on the mortality in autism, genetic prediction of outcome in individuals with a diagnosis, and Uta Frith’s commentary on the concept of “spectrum”. https://pubmed.ncbi.nlm.nih.gov/41773580 https://pubmed.ncbi.nlm.nih.gov/41651809 https://pubmed.ncbi.nlm.nih.gov/41661606 https://archive.ph/fPscR
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