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*, Tiffany House, Benedikt Schoser, Pascal Laforêt, Maudy T.M. Theunissen, Stephan Wenninger, Thomas Hundsberger, Jordi Diaz-Manera, Ans T. van der Ploeg, Nadine A.M.E. van der Beek

*Corresponding author for this work

Research output: Contribution to journal › Comment/Letter to the editor › Academic › peer-review

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