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- FDA approves Ultragenyx gene therapy for glycogen storage disorder; Overton faces confirmation hurdlesThe U.S. Food and Drug Administration has granted approval to Ultragenyx's gene therapy aimed at treating a glycogen storage disorder. This marks a regulatory milestone for the company and the rare…
- Experts call for modernizing rare disease evidence models using FDA plausible mechanism frameworkA recent paper in The Journal of Clinical Investigation argues that the evidentiary approach for rare diseases needs updating. The authors suggest applying the FDA’s plausible mechanism framework,…