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2. Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data

4. Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2)

7. sj-pdf-8-tah-10.1177_20406207211007058 – Supplemental material for Expert opinion on the UK standard of care for haemophilia patients with inhibitors: a modified Delphi consensus study

8. sj-pdf-4-tah-10.1177_20406207211007058 – Supplemental material for Expert opinion on the UK standard of care for haemophilia patients with inhibitors: a modified Delphi consensus study

9. sj-pdf-7-tah-10.1177_20406207211007058 – Supplemental material for Expert opinion on the UK standard of care for haemophilia patients with inhibitors: a modified Delphi consensus study

10. Additional file 1 of Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2)

11. sj-pdf-9-tah-10.1177_20406207211007058 – Supplemental material for Expert opinion on the UK standard of care for haemophilia patients with inhibitors: a modified Delphi consensus study

12. sj-pdf-5-tah-10.1177_20406207211007058 – Supplemental material for Expert opinion on the UK standard of care for haemophilia patients with inhibitors: a modified Delphi consensus study

13. sj-pdf-6-tah-10.1177_20406207211007058 – Supplemental material for Expert opinion on the UK standard of care for haemophilia patients with inhibitors: a modified Delphi consensus study

20. Why I Do Science, Part II

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