Results 61 to 70 of about 39,053 (110)

Broadening applications for intrathecal gene therapy: a case for lysosomal storage diseases. [PDF]

EBioMedicine
eBioMedicine.
europepmc   +1 more source

Editorial: Neuronopathic lysosomal storage diseases - specific neuronal characteristics and therapeutic approaches. [PDF]

Front Mol Neurosci, 2022
Flunkert S, Hutter-Paier B, Sun Y, Kehr J.   +3 more
europepmc   +1 more source

Japanese experience of newborn screening for lysosomal storage diseases and adrenoleukodystrophy. [PDF]

Orphanet J Rare Dis
Onuki T, Tajika M, Sugiyama Y, Shimura M, Ichimoto K, Tanaka T, Nyuzuki H, Kosuga M, Migita O, Ito T, Sasai H, Bo R, Hamada J, Hamazaki T, Sakai N, Inoue T, Nakamura K, Okuyama T, Murayama K.   +18 more
europepmc   +1 more source

Correction to: A systematic review of economic evaluations of enzyme replacement therapy in Lysosomal storage diseases. [PDF]

Cost Eff Resour Alloc, 2022
Katsigianni EI, Petrou P.
europepmc   +1 more source

How to manage hypersensitivity reactions to enzyme replacement therapy in lysosomal storage diseases? [PDF]

Orphanet J Rare Dis
Spataro F, Solimando AG, Desantis V, Vacca A, Di Girolamo A, Ria R.   +5 more
europepmc   +1 more source

Altered blood-brain barrier transport of nanotherapeutics in lysosomal storage diseases. [PDF]

J Control Release, 2022
Solomon M, Loeck M, Silva-Abreu M, Moscoso R, Bautista R, Vigo M, Muro S.   +6 more
europepmc   +1 more source

Secondary accumulation of lyso-platelet activating factors in lysosomal storage diseases. [PDF]

Mol Genet Metab
Kell P, Mishra S, Gray-Edwards HL, Martin DR, Gaudioso A, Ledesma MD, Bongarzone ER, Sands MS, Sidransky E, Ory DS, Jiang X.   +10 more
europepmc   +1 more source

The Antifungal Antibiotic Filipin as a Diagnostic Tool of Cholesterol Alterations in Lysosomal Storage Diseases and Neurodegenerative Disorders. [PDF]

Antibiotics (Basel), 2023
Bruno F, Camuso S, Capuozzo E, Canterini S.   +3 more
europepmc   +1 more source

A systematic review of economic evaluations of enzyme replacement therapy in Lysosomal storage diseases. [PDF]

Cost Eff Resour Alloc, 2022
Katsigianni EI, Petrou P.
europepmc   +1 more source

Lysosomal Storage Diseases—Regulating Neurodegeneration

Journal of Experimental Neuroscience, 2016
Rob U. Onyenwoke, Jay E. Brenman
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