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ArticleOrphanet journal of rare diseases2025

Real-world evidence for Pompe disease remains fragmented. Comment on "A rare partnership: patient community and industry collaboration to shape the impact of real-world evidence on the rare disease ecosystem" by Klein et al.

Michelle E Kruijshaar et al.PubMed ↗Full text ↗Publisher ↗

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Real-world evidence for Pompe disease remains fragmented. Comment on "A rare partnership: patient community and industry collaboration to shape the impact of real-world evidence on the rare disease ecosystem" by Klein et al. | OpenQuestion