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Orphanet Journal of Rare Diseases|March 28, 2025
Epidemiology of gall bladder cancer and its prevalence worldwide: a meta-analysisAli Afzal, Yan-Yan Liu, Amara Noureen, et al.
Orphanet Journal of Rare Diseases|April 1, 2025
Immunization coverage and timeliness of vaccination in young patients with inborn errors of metabolism: a French multicentric studyAnne-Sophie Renous, Lena Damaj, Magali Gorce, et al.
Orphanet Journal of Rare Diseases|April 1, 2025
Like walking through treacle: the experience of fatigue for young people with interstitial lung diseaseCarlee Gilbert, Kate M Bennett, Andrew Bush, et al.
Orphanet Journal of Rare Diseases|November 13, 2025
Exploring medication adherence in Behçet's disease following COVID-19: a mixed-methods studyEmily Arden-Close, Fay Sweeting, Danielle Guy, et al.
Orphanet Journal of Rare Diseases|November 11, 2025
Management of Wilson disease across Europe: an international physician-oriented survey by the ERN-RARE Liver groupFrederik Teicher Kirk, Karina Stubkjær Rewitz, Zoe Mariño, et al.
Orphanet Journal of Rare Diseases|October 31, 2025
Clinical manifestations and treatment of peripheral odontogenic keratocysts: two cases and a literature reviewYu Huang, Lirui Zhang, Xiaohong Yuan, et al.
Orphanet Journal of Rare Diseases|November 11, 2025
Mitigating the impact of study-start delays in clinical trials for rare disorders: insights and lessons from a PKAN trialMarleen M G Bracke, Sjoukje S Polet, Mirjam Plantinga, et al.
Orphanet Journal of Rare Diseases|December 6, 2025
Clinical outcomes of exclusive enzyme therapy (laronidase) in a cohort of patients with mucopolysaccharidosis type INathalie Guffon, Magali Pettazzoni, Nicolas Pangaud, et al.
Orphanet Journal of Rare Diseases|November 13, 2025
Pain assessment and treatment in patients with mucopolysaccharidoses: a French multicentric pediatric studyMélanie Blin, Marine Tardieu, Didier Lacombe, et al.
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