Results 101 to 110 of about 275,755 (180)

Multidisciplinary approach to the assessment and management of children with Fabry disease: Insights from the Chinese Children Genetic Kidney Disease Database. [PDF]

Intractable Rare Dis Res
Wang J, Liu J, Chen J, Zhang A, Mao J, Shen T, Jiang X, Wang M, Tao Y, Zhao B, Wang X, Li Z, Chen A, Chen C, Zhang B, Zhang D, Zhao L, Zhao Y, Bao Y, Bai L, Liu C, Wang F, Hu F, Chen M, Lv X, Sun S, Shen Q, Xu H.   +27 more
europepmc   +1 more source

La Salle and the Historians [PDF]

, 1969
Gum, Ert J
core   +1 more source

Early initiation of enzyme replacement therapy as facilitated by newborn screening improves health outcomes among patients with infantile-onset Pompe disease. [PDF]

Genet Med Open
Desai AK, Rodriguez-Rassi E, Parikh S, Russo RS, Kronn D, Hamm JA, Chang IJ, Ortiz D, McPheron M, Lydigsen H, DeArmey S, Young SP, Kishnani PS.   +12 more
europepmc   +1 more source

First multicenter real-world analysis of switching to next-generation enzyme replacement therapies in late-onset Pompe disease. [PDF]

J Neurol
Mendelsohn DH, Rosenbohm A, Güttsches AK, Kornblum C, Knop KC, Fangerau T, Nguyen-Younossi N, Shahyrova G, Garcia-Angarita N, Schoser B, Wenninger S.   +10 more
europepmc   +1 more source

Comparing the effectiveness of emotion regulation therapy and cognitive behavioral therapy on treatment adherence in hemodialysis patients: A randomized controlled clinical trial. [PDF]

PLoS One
Salimi Arshad Moghaddam Pishkhani SH, Tarrahi MJ, Zargar F.   +2 more
europepmc   +1 more source

Elevated circulating cell-free mitochondrial DNA in fabry disease: insights into inflammatory activation. [PDF]

Front Immunol
Yuan Y, Zhang X, Zhao Y, Wang Y, Huang J, Wang Q, Song X, Deng J, Wang Z, Yuan Y, Zhang W.   +10 more
europepmc   +1 more source

Mapping defect distribution in transparent single-walled carbon nanotube film with electrical resistance tomography. [PDF]

Sci Rep
Minakawa K, Nakada T, Kaneko R, Ikuno T.
europepmc   +1 more source

Clinical outcomes of exclusive enzyme therapy (laronidase) in a cohort of patients with mucopolysaccharidosis type I. [PDF]

Orphanet J Rare Dis
Guffon N, Pettazzoni M, Pangaud N, Reynes N, Le Peillet Feuillet E, Journeau P, Fouilhoux A.   +6 more
europepmc   +1 more source

Long-Term Enzyme Replacement Therapy and Renal Outcomes in Fabry Disease: A Systematic Review and Meta-Analysis. [PDF]

Biomedicines
Chen CY, Li CT, Lin CJ, Shih HM, Liu SJ, Sun FJ, Wu CJ.   +6 more
europepmc   +1 more source

Long-term safety outcomes and patient preferences for home-based intravenous enzyme replacement therapy (ERT) in Pompe disease and Mucopolysaccharidosis Type I (MPS-I): final results of two-year observation. [PDF]

Orphanet J Rare Dis
Toscano A, Musumeci O, Sacchini M, Ravaglia S, Siciliano G, Fiumara A, Verrecchia E, Fischetto R, Crescimanno G, Taurisano R, Sechi A, Gasperini S, Cianci V, Maggi L, Brighina F, Barone R, Cianflone A, Balzarini M, Parini R, Scarpa M.   +19 more
europepmc   +1 more source