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Orphanet Journal of Rare Diseases|April 27, 2021
A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)Fernando de Andrés-Nogales, Encarnación Cruz, Miguel Ángel Calleja, et al.
Orphanet Journal of Rare Diseases|April 11, 2021
Hypertrophy of unaffected cardiomyocytes correlates with severity of cardiomyopathy in female patients with Fabry diseaseCristina Chimenti, Romina Verardo, Andrea Frustaci
Orphanet Journal of Rare Diseases|April 8, 2021
Data silos are undermining drug development and failing rare disease patientsNathan Denton, Monique Molloy, Samantha Charleston, et al.
Orphanet Journal of Rare Diseases|April 8, 2021
Obstructive sleep apnea in Norwegian adults with achondroplasia: a population-based studySvein O Fredwall, Britt Øverland, Hanne Berdal, et al.
Orphanet Journal of Rare Diseases|April 8, 2021
Economic burden in the management of transfusion-dependent thalassaemia patients in Malaysia from a societal perspectiveAsrul Akmal Shafie, Jacqueline Hui Yi Wong, Hishamshah Mohd Ibrahim, et al.
Orphanet Journal of Rare Diseases|April 8, 2021
A framework for the evaluation of patients with congenital facial weaknessBryn D Webb, Irini Manoli, Elizabeth C Engle, et al.
Orphanet Journal of Rare Diseases|April 8, 2021
Clinical and genetic characteristics of hypophosphatasia in Chinese childrenMeijuan Liu, Min Liu, Xuejun Liang, et al.
Orphanet Journal of Rare Diseases|April 20, 2021
The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseasesP K Tandon, Emil D Kakkis
Orphanet Journal of Rare Diseases|March 28, 2013
Experimental designs for small randomised clinical trials: an algorithm for choiceCatherine Cornu, Behrouz Kassai, Roland Fisch, et al.
Orphanet Journal of Rare Diseases|March 23, 2013
Immune response profiling identifies autoantibodies specific to Moyamoya patientsTara K Sigdel, Lorelei D Shoemaker, Rong Chen, et al.
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