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natural history

22nd Jan 2026

Early results from the global X-Linked Hypophosphatemia (XLH) Registry: a plain language summary

28th Aug 2025

Plain language summary: using matched patient data to evaluate omaveloxolone against the natural history of Friedreich ataxia

23rd Apr 2025

Plain language summary of a 1-Year natural history study of Duchenne muscular dystrophy in Chinese individuals

16th Mar 2022

How did transthyretin amyloid cardiomyopathy progress in patients who took placebo in the study ATTR-ACT? A plain language summary

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