[The importance of patient reported outcome measures in Pompe disease].
Mária Judit MolnárViktor MolnárIzabella LászlóMárta SzegediVera VárhegyiZoltán GroszPublished in: Ideggyogyaszati szemle (2021)
For all orphan medicines we emphasize to include patients in a compulsory manner to obtain general and disease-specific multidimensional outcome measures and use them as a quality indicator to monitor treatment effectiveness.