Results 181 to 190 of about 90,357 (220)

Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders. [PDF]

Mol Ther
Kleinboehl EW, Laoharawee K, Jensen JD, Peterson JJ, Slipek NJ, Wick BJ, Johnson MJ, Webber BR, Moriarity BS.   +8 more
europepmc   +1 more source

Drug Repurposing and Lysosomal Storage Disorders: A Trick to Treat. [PDF]

Genes (Basel)
Hay Mele B, Rossetti F, Cubellis MV, Monticelli M, Andreotti G.   +4 more
europepmc   +1 more source

Assessment of the reliability, responsiveness, and meaningfulness of the scale for the assessment and rating of ataxia (SARA) for lysosomal storage disorders. [PDF]

J Neurol
Park J, Bremova-Ertl T, Brands M, Foltan T, Gautschi M, Gissen P, Hahn A, Jones S, Arash-Kaps L, Kolnikova M, Patterson M, Perlman S, Ramaswami U, Reichmannová S, Rohrbach M, Schneider SA, Shaikh A, Sivananthan S, Synofzik M, Walterfarng M, Wibawa P, Martakis K, Manto M.   +22 more
europepmc   +1 more source

Novel pathomechanistic insights into lysosomal storage disorders: how neuron-intrinsic cGAS-STING signaling drives disease progression. [PDF]

Signal Transduct Target Ther
Frosch M, Prinz M.
europepmc   +1 more source

Different diseases, different needs: Patient preferences for gene therapy in lysosomal storage disorders, a probabilistic threshold technique survey. [PDF]

Orphanet J Rare Dis
Corazolla EM, Eskes ECB, Veldwijk J, Brands MMMG, Dekker H, van de Mheen E, Langeveld M, Hollak CEM, Sjouke B.   +8 more
europepmc   +1 more source

Newborn Screening for Lysosomal Storage Disorders

Journal of Pediatric Genetics, 2016
R. Peake, O. Bodamer
semanticscholar   +1 more source

Development and Evaluation of Different Electrospun Cysteamine-Loaded Nanofibrous Webs: A Promising Option for Treating a Rare Lysosomal Storage Disorder

Safaa Omer, Nándor Nagy, Balázs Pinke, László Mészáros, Adrienn Kazsoki, Romána Zelkó   +5 more
openalex   +1 more source

Gene therapy for lysosomal storage disorders

, 2011
Angela Gritti
openalex   +2 more sources

Measuring health related quality of life (HRQoL) in Lysosomal Storage Disorders (LSDs): a rapid scoping review of available tools and domains. [PDF]

Orphanet J Rare Dis
McDool E, Powell P, Carlton J.
europepmc   +1 more source

Mitochondrial Dysfunction and Neurodegeneration in Lysosomal Storage Disorders.

Trends in Molecular Medicine, 2017
N. Plotegher, M. Duchen
semanticscholar   +1 more source