Pentosan Polysulfate: Oral Versus Subcutaneous Injection in Mucopolysaccharidosis Type I Dogs von Simonaro, Calogera M, Tomatsu, Shunji, Sikora, Tracy, Kubaski, Francyne, Frohbergh, Michael, Guevara, Johana M, Wang, Raymond Y, Vera, Moin, Kang, Jennifer L, Smith, Lachlan J, Schuchman, Edward H, Haskins, Mark E

Volltext

New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy von Schuchman, Edward H., Ledesma, Maria D., Simonaro, Calogera M.

Volltext

Pentosan polysulfate: a novel therapy for the mucopolysaccharidoses von Schuchman, Edward H, Ge, Yi, Lai, Alon, Borisov, Yury, Faillace, Meghan, Eliyahu, Efrat, He, Xingxuan, Iatridis, James, Vlassara, Helen, Striker, Gary, Simonaro, Calogera M

Volltext

Involvement of the Toll-like receptor 4 pathway and use of TNF-α antagonists for treatment of the mucopolysaccharidoses von Simonaro, Calogera M, Ge, Yi, Eliyahu, Efrat, He, Xingxuan, Jepsen, Karl J, Schuchman, Edward H

Volltext

Dose responsive effects of subcutaneous pentosan polysulfate injection in mucopolysaccharidosis type VI rats and comparison to oral treatment von Frohbergh, Michael, Ge, Yi, Meng, Fanli, Karabul, Nesrin, Solyom, Alexander, Lai, Alon, Iatridis, James, Schuchman, Edward H, Simonaro, Calogera M

Volltext

Anti-TNF-alpha therapy enhances the effects of enzyme replacement therapy in rats with mucopolysaccharidosis type VI von Eliyahu, Efrat, Wolfson, Theodore, Ge, Yi, Jepsen, Karl J, Schuchman, Edward H, Simonaro, Calogera M

Volltext

Acid Ceramidase Protects Against Hepatic Ischemia/Reperfusion Injury by Modulating Sphingolipid Metabolism and Reducing Inflammation and Oxidative Stress von Jiang, Yuan, He, Xingxuan, Simonaro, Calogera M., Yi, Bin, Schuchman, Edward H.

Volltext

Systemic ceramide accumulation leads to severe and varied pathological consequences von Alayoubi, Abdulfatah M., Wang, James C. M., Au, Bryan C. Y., Carpentier, Stéphane, Garcia, Virginie, Dworski, Shaalee, El‐Ghamrasni, Samah, Kirouac, Kevin N., Exertier, Mathilde J., Xiong, Zi Jian, Privé, Gilbert G., Simonaro, Calogera M., Casas, Josefina, Fabrias, Gemma, Schuchman, Edward H., Turner, Patricia V., Hakem, Razqallah, Levade, Thierry, Medin, Jeffrey A.

Volltext

Acid ceramidase maintains the chondrogenic phenotype of expanded primary chondrocytes and improves the chondrogenic differentiation of bone marrow-derived mesenchymal stem cells von Simonaro, Calogera M, Sachot, Sylvain, Ge, Yi, He, Xingxuan, Deangelis, Victor A, Eliyahu, Efrat, Leong, Daniel J, Sun, Hui B, Mason, Jeffrey B, Haskins, Mark E, Richardson, Dean W, Schuchman, Edward H

Volltext

Endocannabinoid receptor 2 is a potential biomarker and therapeutic target for the lysosomal storage disorders von Simonaro, Calogera M., Yasuda, Makiko, Schuchman, Edward H.

Volltext

Joint and bone disease in mucopolysaccharidoses VI and VII : Identification of new therapeutic targets and biomarkers using animal models von SIMONARO, Calogera M, D'ANGELO, Marina, HASKINS, Mark E, SCHUCHMAN, Edward H

Volltext

The Demographics and Distribution of Type B Niemann-Pick Disease: Novel Mutations Lead to New Genotype/Phenotype Correlations von Simonaro, Calogera M., Desnick, Robert J., McGovern, Margaret M., Wasserstein, Melissa P., Schuchman, Edward H.

Volltext

Lysosomes, Lysosomal Storage Diseases, and Inflammation von Simonaro, Calogera M.

Volltext

Treatment with pentosan polysulphate in patients with MPS I: results from an open label, randomized, monocentric phase II study von Hennermann, Julia B., Gökce, Seyfullah, Solyom, Alexander, Mengel, Eugen, Schuchman, Edward H., Simonaro, Calogera M.

Volltext

Mechanism of Glycosaminoglycan-Mediated Bone and Joint Disease: Implications for the Mucopolysaccharidoses and Other Connective Tissue Diseases von Simonaro, Calogera M, D'Angelo, Marina, He, Xingxuan, Eliyahu, Efrat, Shtraizent, Nataly, Haskins, Mark E, Schuchman, Edward H

Volltext

A New Fluorescent Method to Detect Sulfamidase Activity in Blood, Tissue Extracts and Dried Blood Spots von He, Xingxuan, Schuchman, Edward H., Simonaro, Calogera M.

Volltext

Insertional Mutagenesis of the Mouse Acid Ceramidase Gene Leads to Early Embryonic Lethality in Homozygotes and Progressive Lipid Storage Disease in Heterozygotes von Li, Chi-Ming, Park, Jae-Ho, Simonaro, Calogera M., He, Xingxuan, Gordon, Ronald E., Friedman, Adriana-Haimovitz, Ehleiter, Desiree, Paris, Francois, Manova, Katia, Hepbiloikler, Stefan, Fuks, Zvi, Sandhoff, Konrad, Kolesnick, Richard, Schuchman, Edward H.

Volltext

Enzyme replacement therapy for Farber disease: Proof-of-concept studies in cells and mice von He, Xingxuan, Dworski, Shaalee, Zhu, Changzhi, DeAngelis, Victor, Solyom, Alex, Medin, Jeffrey A., Simonaro, Calogera M., Schuchman, Edward H.

Volltext

Pentosan Polysulfate Treatment of Mucopolysaccharidosis Type IIIA Mice von Guo, Ningning, DeAngelis, Victor, Zhu, Changzhi, Schuchman, Edward H., Simonaro, Calogera M.

Volltext

Identification and characterization of eight novel SMPD1 mutations causing types A and B Niemann-Pick disease von Desnick, Jonathan P, Kim, Jungmin, He, Xingxuan, Wasserstein, Melissa P, Simonaro, Calogera M, Schuchman, Edward H

Volltext