Zoekresultaten - "enzyme replacement therapy"

Enzyme replacement therapy: efficacy and limitations door Daniela Concolino, Federica Deodato, Rossella Parini

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Mucopolysaccharidosis type II: Enzyme Replacement Therapy Efficiency door Nato D. Vashakmadze, Leyla S. Namazova-Baranova, Natalia V. Zhurkova, Ekaterina Yu. Zakharova, Grigory V. Revunenkov, Tina V. Lobjanidze, Marina A. Babaikina

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An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy door Goker-Alpan O, Longo N, McDonald M, Shankar SP, Schiffmann R, Chang P, Shen Y, Pano A

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Lysosomal acid lipase deficiency: analysis of enzyme replacement therapy door N.A. Polyanskaya, A.A. Gorbunova, E.B. Pavlinova, O.A. Savchenko, I.A. Kirshina, M.E. Bagaeva, T.V. Strokova

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Enzyme replacement therapy for Fabry disease: some answers but more questions door Majid Alfadhel, Sandra Sirrs

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Clinical features and enzyme replacement therapy in 10 children with Fabry disease door Qian Li, Qian Li, Jing Wang, Jing Wang, Minle Tian, Zhenle Yang, Zhenle Yang, Lichun Yu, Lichun Yu, Suwen Liu, Suwen Liu, Cong Wang, Cong Wang, Xiaoyuan Wang, Xiaoyuan Wang, Shuzhen Sun, Shuzhen Sun

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Cognitive Outcome of Infants with Pompe Disease Receiving Enzyme-Replacement Therapy door J Gordon Millichap

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Allergic Reactions at Enzyme Replacement Therapy in Children with Mucopolysaccharidosis Type II door Julia G. Levina, Nato D. Vashakmadze, Leyla S. Namazova-Baranova, Elena A. Vishneva, Natalia V. Zhurkova, Kamilla E. Efendieva, Anna A. Alekseeva, Vera G. Kalugina

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Dental effects of enzyme replacement therapy in case of childhood-type hypophosphatasia door Rena Okawa, Kazuma Kokomoto, Kazuhiko Nakano

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The COVID-19 Pandemic and Enzyme Replacement Therapy in Lysosomal Storage Disorders door Asburçe Olgaç, Çiğdem Seher Kasapkara, Burak Açıkel, Yılmaz Yıldız, Gülhan Karakaya Molla, Mustafa Kılıç

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Changing Times for CLN2 Disease: The Era of Enzyme Replacement Therapy door Specchio N, Pietrafusa N, Trivisano M

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Dose selection for biological enzyme replacement therapy indicated for inborn errors of metabolism door Yuen Yi Hon, Jie Wang, Henrietta Abodakpi, Anand Balakrishnan, Michael Pacanowski, Sushanta Chakder, Patroula Smpokou, Kathleen Donohue, Yow‐Ming C. Wang

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Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease door Lina Fiege, Ibrahim Duran, Thorsten Marquardt

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Enzyme Replacement Therapy with Idursulfase in Patients with Mucopolysaccharidosis Type II: Literature Review door Nato D. Vashakmadze, Natalya V. Zhurkova, Olga B. Gordeeva, Elena V. Komarova, Tatyana E. Privalova, Anastasya I. Rykunova, Marina А. Babaykina

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Cardiovascular findings and effects of enzyme replacement therapy in patients with mucopolysaccharidosis type VI door Emine Azak, Mehmet Gündüz

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EVALUATION OF ENZYME REPLACEMENT THERAPY EFFECTIVENESS IN CHILDREN WITH GAUCHER'S DISEASE ACCORDING TO THE INTERNATIONAL STUDIES door O. S. Gundobina, G. B. Movsisyan, L. S. Namazova-Baranova

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Modeling the effect of enzyme replacement therapy on life-threatening complications in patients with Fabry disease door V. I. Ignatyeva, S. V. Moiseev, N. M. Bulanov, E. A. Karovajkina, A S. Moiseev

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Considerations for evaluating the effectiveness and long-term outcome of enzyme replacement therapy in Pompe disease door Chong Kun Cheon

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Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy was underpowered door Ortiz A, Sanchez-Niño MD

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Implementation and results of a risk-sharing scheme for enzyme replacement therapy in lysosomal storage diseases door María Dolores Edo-Solsona, José Luis Poveda-Andrés, Isidro Vitoria-Miñana

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