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Addressing psychosocial vulnerability in rare diseases: a call to action from a European expert consensus study.
Smits RM, Aerts A, Angelov T, Bentele M, de Blaauw I, Dellenmark-Blom M, Duijts SFA, Fjermestad K, Franscini M, Grano C, Hendriks S, Inhestern L, Kenny T, von der Lippe C, Oomen L, Rake JP, Rietman AB, van Rooij IALM, Verhaak CM, Voet NBM, Walton H, van Zelst-Stams W, Kwakkenbos L. Smits RM, et al. Among authors: verhaak cm. Orphanet J Rare Dis. 2025 Oct 27;20(1):543. doi: 10.1186/s13023-025-04017-3. Orphanet J Rare Dis. 2025. PMID: 41146214 Free PMC article.
Complications After Surgery for Anorectal Malformations: An ARM-Net consortium Registry Study.
van der Steeg HJJ, Hageman IC, Morandi A, Amerstorfer EE, Sloots CEJ, Jenetzky E, Stenström P, Samuk I, Fanjul M, Midrio P, Schmiedeke E, Iacobelli BD, de Blaauw I, van Rooij IALM; ARM-Net Consortium. van der Steeg HJJ, et al. J Pediatr Surg. 2025 Sep;60(9):162403. doi: 10.1016/j.jpedsurg.2025.162403. Epub 2025 Jun 6. J Pediatr Surg. 2025. PMID: 40482809 Free article.
Phase 2b program with sonlicromanol in patients with mitochondrial disease due to m.3243A>G mutation.
Smeitink J, van Es J, Bosman B, Janssen MCH, Klopstock T, Gorman G, Vissing J, Ruiterkamp G, Edgar CJ, Abbink EJ, van Maanen R, Pogoryelova O, Stendel C, Bischoff A, Karin I, Munshi M, Kümmel A, Burgert L, Verhaak C, Renkema H. Smeitink J, et al. Among authors: verhaak c. Brain. 2025 Mar 6;148(3):896-907. doi: 10.1093/brain/awae277. Brain. 2025. PMID: 39501914 Free PMC article. Clinical Trial.
The European Anorectal Malformation Network (ARM-Net) patient registry: 10-year review of clinical and surgical characteristics.
Hageman IC, Midrio P, van der Steeg HJJ, Jenetzky E, Iacobelli BD, Morandi A, Sloots CEJ, Schmiedeke E, Broens PMA, Fascetti Leon F, Çavuşoğlu YH, Gorter RR, Trajanovska M, King SK, Aminoff D, Schwarzer N, Haanen M, de Blaauw I, van Rooij IALM; ARM-Net Consortium. Hageman IC, et al. Br J Surg. 2024 Jan 31;111(2):znae019. doi: 10.1093/bjs/znae019. Br J Surg. 2024. PMID: 38364059 Free PMC article. Review. No abstract available.
Health-related quality of life in children with congenital vascular malformations.
Bouwman FCM, Verhaak C, de Blaauw I, Kool LJS, Loo DMWMT, van Rooij IALM, van der Vleuten CJM, Botden SMBI, Verhoeven BH. Bouwman FCM, et al. Among authors: verhaak c. Eur J Pediatr. 2023 Nov;182(11):5067-5077. doi: 10.1007/s00431-023-05166-y. Epub 2023 Sep 4. Eur J Pediatr. 2023. PMID: 37665335 Free PMC article.
120 results