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A rare partnership: patient community and industry collaboration to shape the impact of real-world evidence on the rare disease ecosystem.
Klein TL, Bender J, Bolton S, Collin-Histed T, Daher A, De Baere L, Dong D, Hopkin J, Johnson J, Lai T, Pavlou M, Schaller T, Žnidar I. Klein TL, et al. Among authors: znidar i. Orphanet J Rare Dis. 2024 Jul 10;19(1):262. doi: 10.1186/s13023-024-03262-2. Orphanet J Rare Dis. 2024. PMID: 38987844 Free PMC article. Review.
Transition of patients with Gaucher disease type 1 from pediatric to adult care: results from two international surveys of patients and health care professionals.
Stepien KM, Žnidar I, Kieć-Wilk B, Jones A, Castillo-García D, Abdelwahab M, Revel-Vilk S, Lineham E, Hughes D, Ramaswami U, Collin-Histed T. Stepien KM, et al. Among authors: znidar i. Front Pediatr. 2024 Aug 27;12:1439236. doi: 10.3389/fped.2024.1439236. eCollection 2024. Front Pediatr. 2024. PMID: 39346636 Free PMC article.
The road to biosimilars in rare diseases - ongoing lessons from Gaucher disease.
Drelichman G, Castañeda-Hernández G, Cem Ar M, Dragosky M, Garcia R, Lee H, Moiseev S, Naderi M, Rosenbaum H, Žnidar I, Zuluaga AF, Freisens S, Mistry PK. Drelichman G, et al. Among authors: znidar i. Am J Hematol. 2020 Mar;95(3):233-237. doi: 10.1002/ajh.25701. Epub 2019 Dec 23. Am J Hematol. 2020. PMID: 31816110 Free PMC article. Review. No abstract available.