Results 91 to 100 of about 20,963 (163)

Treatment-related benefit and satisfaction in patients with Fabry disease in France: insight into patients' expectations and preferences from the prospective, non-interventional SATIS-Fab study. [PDF]

Orphanet J Rare Dis
Lidove O, Masseau A, Pugnet G, Lacombe D, Dussol B, Bekri S, Hagège A, Martinez C, Fardini Y, Fouilhoux A, Noël E.   +10 more
europepmc   +1 more source

CSF GAG non-reducing ends in MPS IH after peripheral and brain-penetrating therapies: A model comparing IV non-targeted ERT and HCT. [PDF]

Mol Ther Adv
Lund TC, Wagner JE, Long S, Gupta AO, Orchard PJ, Braunlin E, Whitley CB, Eisengart JB.   +7 more
europepmc   +1 more source

Gastrointestinal manifestations and enzyme replacement therapy in late-onset Pompe disease: insights from a cross-sectional analysis. [PDF]

Orphanet J Rare Dis
Liu X, Li X, Yao X, Zhu G, Guo X, He W, Hu L, Yang G.   +7 more
europepmc   +1 more source

Management of life-threatening anaphylaxis to enzyme replacement therapy in an infant with Pompe disease: a case report and literature review. [PDF]

Front Allergy
Zhou J, Wang Q, Zhang H, Wang Y, Wang Z, Yang F, Yang S.   +6 more
europepmc   +1 more source

Estrogen deficiency and risk of hearing loss in pediatric Turner syndrome. [PDF]

J Clin Invest
Huang Y, Liang L, Ye Y, Zhang L, Ling L, Meng Z, Liu W, Guo J, Liu Z, Zhao Z, Zhang Z, Si Y.   +11 more
europepmc   +1 more source

Teacher Reflections on ERT

Teacher Reflections on ERT
The Sojo University English teachers of first-year students taught a new, online curriculum during the 2020 fall semester. All nine teachers reflected on the semester using the DEAL model. These reflections focused on five areas that are specific to the role of the teacher.
openaire  

Use of nonsteroidal mineralocorticoid receptor antagonist in chronic kidney disease: A case report of a patient with Fabry disease in his mid-20s. [PDF]

J Int Med Res
Tian GQ, Yang P, Liao ZC, Xiao W, Li J, Song B.   +5 more
europepmc   +1 more source

Delayed diagnosis of mucopolysaccharidosis type I in a patient with spinopelvic instability, short stature, and skeletal dysplasia. [PDF]

JBMR Plus
Windels O, Lindschau M, Boschann F, Oheim R, Muschol N.   +4 more
europepmc   +1 more source

Multidimensional assessment of diaphragmatic dysfunction in late-onset Pompe disease: a prospective cohort study. [PDF]

Orphanet J Rare Dis
Hernández-Voth A, Sayas-Catalán J, Corral-Blanco M, Jiménez-Gómez M, Carvajal-Cuesta G, Domínguez-González C, Bermejo-Guerrero L, Martín-Jiménez P, Villena-Garrido V.   +8 more
europepmc   +1 more source

Multi-session CBM-I for social anxiety: examining psychopathology, cognitive, neural, and psychophysiological effects in a randomized controlled trial. [PDF]

Transl Psychiatry
Abado E, Kunna M, Würtz F, Laflör L, Blank L, Blackwell SE, Salemink E, Adolph D, Dietel FA, Wolf OT, Margraf J, Woud ML.   +11 more
europepmc   +1 more source