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Expert Review of Medical Devices|August 22, 2019
Intermediate outcomes of transcatheter pulmonary valve replacement with the Edwards Sapien 3 valve - German experienceAnja Lehner, Tsvetina Dashkalova, Sarah Ulrich, et al.
Molecular Genetics and Metabolism|November 13, 2025
Clinical characteristics and real-world outcomes in patients with mucopolysaccharidosis II over 18 years: final report of the Hunter Outcome SurveyJoseph Muenzer, Jaco Botha, Hernan Amartino, et al.
Orphanet Journal of Rare Diseases|June 20, 2022
Twenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registryMichael Beck, Uma Ramaswami, Elizabeth Hernberg-Ståhl, et al.
Orphanet Journal of Rare Diseases|July 1, 2016
Multicenter Female Fabry Study (MFFS) - clinical survey on current treatment of females with Fabry diseaseMalte Lenders, Julia B Hennermann, Christine Kurschat, et al.
Journal of the Endocrine Society|December 22, 2017
Homozygous Resistance to Thyroid Hormone <i>β</i>: Can Combined Antithyroid Drug and Triiodothyroacetic Acid Treatment Prevent Cardiac Failure?Carla Moran, Abdelhadi M Habeb, George J Kahaly, et al.
Clinical Pharmacology and Therapeutics|March 22, 2020
Treatment of Fabry's Disease With Migalastat: Outcome From a Prospective Observational Multicenter Study (FAMOUS)Malte Lenders, Peter Nordbeck, Christine Kurschat, et al.
European Heart Journal. Cardiovascular Pharmacotherapy|May 5, 2022
Treatment of Fabry Disease management with migalastat-outcome from a prospective 24 months observational multicenter study (FAMOUS)Malte Lenders, Peter Nordbeck, Christine Kurschat, et al.
Orphanet Journal of Rare Diseases|November 9, 2011
Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare diseaseMaurizio Scarpa, Zsuzsanna Almássy, Michael Beck, et al.
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