Journal: PLoS ONE

Article Title: Odiparcil, a potential glycosaminoglycans clearance therapy in mucopolysaccharidosis VI—Evidence from in vitro and in vivo models

doi: 10.1371/journal.pone.0233032

Figure Lengend Snippet: A, B [CS-56] detection of total cellular (A) and intracellular (B) CS in fibroblasts from MPS VI patient, C. high magnification of B. D, E and F. Partial co-localization of intracellular CS (D, imaging conditions chosen for best display of perinuclear CS staining) with GLG1 (Golgi) marker (E) seen on the overlay (F). G, H and I. Partial co-localization of intracellular CS (G, imaging conditions chosen for best display of dotty cytoplasmic CS staining) with LAMP1 (lysosomes) marker (H) seen on the overlay (I). Scale bars: A, B– 50 μm, C-I– 20 μm.

Article Snippet: Primary skin fibroblasts from MPS VI patients GM00538 and GM02572 were obtained from the NIGMS Human Genetic Cell Repository at the Coriell Institute for Medical Research.

Techniques: Imaging, Staining, Marker

Journal: PLoS ONE

Article Title: Odiparcil, a potential glycosaminoglycans clearance therapy in mucopolysaccharidosis VI—Evidence from in vitro and in vivo models

doi: 10.1371/journal.pone.0233032

Figure Lengend Snippet: A, B. Effect of odiparcil treatment on intracellular CS in MPS VI patient fibroblasts GM00538 in growing cells culture conditions (A) and in confluent cells culture conditions (B). C, D. High magnification of intracellular CS staining in MPS VI patient fibroblasts GM00538, no odiparcil treatment (C) and 10 μM odiparcil (D). E. Stimulation of excretion of sulphated GAG in cell culture medium by odiparcil treatment in MPS VI donors GM00538 and GM02572. F, G. Quantification of the effect of odiparcil on the intracellular CS in MPS VI patient fibroblasts GM00538 in growing cells culture conditions (F) and in confluent cells culture conditions (G). Scale bar: C, D– 50 μm. E-G—Data represented as mean ± SEM.

Article Snippet: Primary skin fibroblasts from MPS VI patients GM00538 and GM02572 were obtained from the NIGMS Human Genetic Cell Repository at the Coriell Institute for Medical Research.

Techniques: Staining, Cell Culture

Journal: Orphanet Journal of Rare Diseases

Article Title: Registries for orphan drugs: generating evidence or marketing tools?

doi: 10.1186/s13023-020-01519-0

Figure Lengend Snippet: Overview of the LSD disease registries sponsored by pharmaceutical industry

Article Snippet: MPS VI , Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP) , 2005 , BioMarin Pharmaceutical , 200.

Techniques: