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DISEASE BURDEN AND DISEASE PROGRESSION OF YOUNG ADULT PATIENTS WITH AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE
DISEASE BURDEN AND DISEASE PROGRESSION OF YOUNG ADULT PATIENTS WITH AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE
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DISEASE BURDEN AND DISEASE PROGRESSION OF YOUNG ADULT PATIENTS WITH AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE

Davenport, Eric ; Zhou, Xiaolei ; Wang, Jinyi ; Nunna, Sasikiran ; Oberdhan, Dorothee ; Fernandes, Ancilla ; Mekahli, Djalila

AMERICAN JOURNAL OF KIDNEY DISEASES, 2023, Vol.81 (4), p.S102-S102

W B SAUNDERS CO-ELSEVIER INC

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The Clinico-Genomic Landscape of Induction Failure in Childhood and Young Adult T-Cell Acute Lymphoblastic Leukemia (T-ALL)
The Clinico-Genomic Landscape of Induction Failure in Childhood and Young Adult T-Cell Acute Lymphoblastic Leukemia (T-ALL)
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The Clinico-Genomic Landscape of Induction Failure in Childhood and Young Adult T-Cell Acute Lymphoblastic Leukemia (T-ALL)

O'Connor, David ; Demeulemeester, Jonas ; Conde, Lucia ; Kirkwood, Amy A ; Fung, Kent ; Papaleonidopoulou, Foteini ; Bloye, Gianna ; Farah, Nadine ; Rahman, Sunniyat ; Hancock, Jeremy ; Bateman, Caroline ; Inglott, Sarah ; Mee, Jon ; Herrero, Javier ; Van Loo, Peter ; Moorman, Anthony ; Vora, Ajay ; Mansour, Marc R

BLOOD, 2022, Vol.140, p.1720-1721

AMER SOC HEMATOLOGY

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3
Evaluation of thigh muscle fat fraction with quantitative MRI in 24 adult LGMDR12 patients over 2 years of follow-up
Evaluation of thigh muscle fat fraction with quantitative MRI in 24 adult LGMDR12 patients over 2 years of follow-up
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Evaluation of thigh muscle fat fraction with quantitative MRI in 24 adult LGMDR12 patients over 2 years of follow-up

De Wel, B ; Huysmans, L ; Peeters, R ; Goosens, V ; Ghysels, S ; Byloos, K ; Putzeys, G ; D'Hondt, A ; De Bleecker, J ; Dupont, P ; Maes, F ; Claeys, K

NEUROMUSCULAR DISORDERS, 2022, Vol.32, p.S118-S118

PERGAMON-ELSEVIER SCIENCE LTD

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4
Two-years prospective natural history study in 29 adult LGMDR12 patients: Clinical and radiological outcome measures
Two-years prospective natural history study in 29 adult LGMDR12 patients: Clinical and radiological outcome measures
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Two-years prospective natural history study in 29 adult LGMDR12 patients: Clinical and radiological outcome measures

De Wel, B ; Huysmans, L ; Peeters, R ; D'Hondt, A ; Goosens, V ; Ghysels, S ; Byloos, K ; Putzeys, G ; De Bleecker, J ; Maes, F ; Dupont, P ; Claeys, K

NEUROMUSCULAR DISORDERS, 2021, Vol.31, p.S106-S106

PERGAMON-ELSEVIER SCIENCE LTD

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5
Overlapping and divergent hepatic and lipoprotein phenotypes in untreated adults with acid sphingomyelinase deficiency versus untreated adults with Gaucher disease from two pivotal clinical trials
Overlapping and divergent hepatic and lipoprotein phenotypes in untreated adults with acid sphingomyelinase deficiency versus untreated adults with Gaucher disease from two pivotal clinical trials
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Overlapping and divergent hepatic and lipoprotein phenotypes in untreated adults with acid sphingomyelinase deficiency versus untreated adults with Gaucher disease from two pivotal clinical trials

Cassiman, David ; Mistry, Pramod K ; Jones, Simon A ; Lachmann, Robin ; Lukina, Elena ; Prada, Carlos E ; Thurberg, Beth L ; Wasserstein, Melissa P ; Foster, Meredith C ; Patel, Reema M ; Ribes, Manuel ; Underhill, Lisa H ; Peterschmitt, M Judith

MOLECULAR GENETICS AND METABOLISM, 2022, Vol.135 (2), p.S29-S29

ACADEMIC PRESS INC ELSEVIER SCIENCE

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6
Exploration of muscle MR imaging and clinical outcome measures in adults with Becker muscular dystrophy
Exploration of muscle MR imaging and clinical outcome measures in adults with Becker muscular dystrophy
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Exploration of muscle MR imaging and clinical outcome measures in adults with Becker muscular dystrophy

De Wel, B ; Iterbeke, L ; Huysmans, L ; Peeters, R ; Goosens, V ; Ghysels, S ; Byloos, K ; Putzeys, G ; Dubuisson, N ; van den Bergh, P ; Van Parijs, V ; Remiche, G ; Maes, F ; Dupont, P ; Claeys, K

NEUROMUSCULAR DISORDERS, 2023, Vol.33, p.S146-S146

PERGAMON-ELSEVIER SCIENCE LTD

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7
Olipudase alfa enzyme replacement therapy reverses interstitial lung disease in adults with acid sphingomyelinase deficiency: Long-term pulmonary outcomes of the ASCEND trial
Olipudase alfa enzyme replacement therapy reverses interstitial lung disease in adults with acid sphingomyelinase deficiency: Long-term pulmonary outcomes of the ASCEND trial
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Olipudase alfa enzyme replacement therapy reverses interstitial lung disease in adults with acid sphingomyelinase deficiency: Long-term pulmonary outcomes of the ASCEND trial

Wasserstein, Melissa ; Bonella, Francesco ; Giugliani, Roberto ; Lachmann, Robin ; Noth, Imre ; Scarpa, Maurizio ; Wuyts, Wim A ; Aguiar, Mario ; Armstrong, Nicole ; Kumar, Monica ; Berger, Kenneth

MOLECULAR GENETICS AND METABOLISM, 2024, Vol.141 (2)

ACADEMIC PRESS INC ELSEVIER SCIENCE

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8
Impact of Cytoreductive Drugs upon Outcomes in a Contemporary Cohort of Adolescent and Young Adults with Essential Thrombocythemia and Polycythemia Vera
Impact of Cytoreductive Drugs upon Outcomes in a Contemporary Cohort of Adolescent and Young Adults with Essential Thrombocythemia and Polycythemia Vera
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Impact of Cytoreductive Drugs upon Outcomes in a Contemporary Cohort of Adolescent and Young Adults with Essential Thrombocythemia and Polycythemia Vera

Beauverd, Yan ; Ianotto, Jean-Christophe ; Thaw, Kyaw Htin ; Sobas, Marta ; Sadjadian, Parvis ; Curto-Garcia, Natalia ; Shih, Lee-Yung ; Devos, Timothy ; Krochmalczyk, Dorota ; Galli, Serena ; Bieniaszewska, Maria ; Seferynska, Ilona ; McMullin, Mary Frances ; Armatys, Anna ; Spalek, Adrianna ; Waclaw, Joanna ; Zdrenghea, Mihnea Tudor ; Legros, Laurence ; Girodon, Francois ; Lewandowski, Krzysztof ; Angona Figueras, Anna ; Samuelsson, Jan ; Abuin Blanco, Aitor ; Cony-Makhoul, Pascale ; Collins, Angela ; James, Chloe ; Kusec, Rajko ; Lauermannova, Marie ; Soledad Noya, Maria ; Skowronek, Malgorzata ; Szukalski, Lukasz ; Szmigielska-Kaplon, Anna ; Wondergem, Marielle ; Dudchenko, Iryna ; Gora-Tybor, Joanna ; Laribi, Kamel ; De Nalecz, Anna Kulikowska ; Demory, Jean-Loup ; Le Du, Katell ; Zweegman, Sonja ; Besses Raebel, Carlos ; Skoda, Radek C ; Giraudier, Stephane ; Griesshammer, Martin ; Kiladjian, Jean-Jacques ; Harrison, Claire N

BLOOD, 2023, Vol.142

AMER SOC HEMATOLOGY

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9
Effect size analysis of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL
Effect size analysis of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL
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Effect size analysis of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL

Diaz-Manera, J ; Bratkovic, D ; Byrne, B ; Claeys, K ; Dimachkie, M ; Kushlaf, H ; Kishnani, P ; Laforet, P ; Mozaffar, T ; Roberts, M ; Toscano, A ; Castelli, J ; Raza, S ; Holdbrook, F ; Das, S Sitaraman ; Wasfi, Y ; Schoser, B

NEUROMUSCULAR DISORDERS, 2023, Vol.33, p.S151-S151

PERGAMON-ELSEVIER SCIENCE LTD

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10
Introducing dynamic balance assessment in Adult Spinal Deformity
Introducing dynamic balance assessment in Adult Spinal Deformity
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Introducing dynamic balance assessment in Adult Spinal Deformity

Moke, Lieven ; Severijns, Pieter ; Doskarova, Tereza ; Molenaers, Guy ; Desloovere, Kaat ; Scheys, Lennart

Gait & Posture, 2016, Vol.49 (153)

Butterworth-Heinemann

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