Rare lysosomal disease registries: lessons learned over three decades of real-world evidence von Mistry, P K, Kishnani, P, Wanner, C, Dong, D, Bender, J, Batista, J L, Foster, J

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Management of non-neuronopathic Gaucher disease with special reference to pregnancy, splenectomy, bisphosphonate therapy, use of biomarkers and bone disease monitoring von Cox, T. M., Aerts, J. M. F. G., Belmatoug, N., Cappellini, M. D., vom Dahl, S., Goldblatt, J., Grabowski, G. A., Hollak, C. E. M., Hwu, P., Maas, M., Martins, A. M., Mistry, P. K., Pastores, G. M., Tylki-Szymanska, A., Yee, J., Weinreb, N.

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Ulcerative colitis has an aggressive course after orthotopic liver transplantation for primary sclerosing cholangitis von Papatheodoridis, G V, Hamilton, M, Mistry, P K, Davidson, B, Rolles, K, Burroughs, A K

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Long‐Term Outcomes of Liver Transplantation in Type 1 Gaucher Disease von Ayto, R. M., Hughes, D. A., Jeevaratnam, P., Rolles, K., Burroughs, A. K., Mistry, P. K., Mehta, A. B., Pastores, G. M.

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Oxidative-phosphorylation defects in liver of patients with Wilson's disease von Gu, M, Cooper, JM, Butler, P, Walker, AP, Mistry, PK, Dooley, JS, Schapira, AHV

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Outcome of orthotopic liver transplantation in patients with haemophilia von Gordon, F H, Mistry, P K, Sabin, C A, Lee, C A

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Enzyme replacement therapy and bony changes in Egyptian paediatric Gaucher disease patients von El‐Beshlawy, A., Ragab, L., Youssry, I., Yakout, K., El‐Kiki, H., Eid, K., Mansour, I. M., El‐Hamid, S. Abd, Yang, M., Mistry, P. K.

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Laboratory parameters associated with prolonged hospital length of stay in COVID-19 patients in Johannesburg, South Africa von Pillai, J, Mistry, P P K, Le Roux, D A, Motaung, K S C, Mokgatle, M, Gaylard, P, Cengiz, N, Basu, D

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Therapeutic delivery of proteins to macrophages: implications for treatment of Gaucher's disease von Mistry, Pramod K, Wraight, E Philip, Cox, Timothy M

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Treatment of Gaucher's disease with OGT 918 von Mistry, PK

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Recombinant macrophage targeted enzyme replacement therapy for Gaucher disease in India von Nagral, A., Mewawalla, P., Jagadeesh, S., Kabra, M., Phadke, S. R., Verma, I. C., Puri, R. D., Gupta, N., Kishnani, P. S., Mistry, P. K.

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Glucosylsphingosine Promotes α-Synuclein Pathology in Mutant GBA-Associated Parkinson's Disease von Taguchi, Yumiko V, Liu, Jun, Ruan, Jiapeng, Pacheco, Joshua, Zhang, Xiaokui, Abbasi, Justin, Keutzer, Joan, Mistry, Pramod K, Chandra, Sreeganga S

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Risk factors for fractures and avascular osteonecrosis in type 1 Gaucher disease: A study from the International Collaborative Gaucher Group (ICGG) Gaucher Registry von Khan, Aneal, Hangartner, Thomas, Weinreb, Neal J, Taylor, John S, Mistry, Pramod K

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Blood and graft eosinophils in acute cellular rejection of liver allografts von Nagral, A, Quaglia, A, Sabin, C.A, Dhillon, A.P, Bearcroft, C.P, Millar, A, Davies, S.E, Mistry, P.K, Davidson, B.R, Rolles, K, Burroughs, A.K

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Genetic diagnosis of Gaucher's disease von Mistry, P.K., Smith, S.J., Ali, M., Cox, T.M., Hatton, C.S.R., McIntyre, N.

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The association between free fatty acid concentrations and triglyceride-rich lipoproteins in the post-prandial state is altered by a common deletion polymorphism of the apo B signa... von Byrne, C.D., Wareham, N.J., Mistry, P.K., Phillips, D.I.W., Martensz, N.D., Halsall, D., Talmud, P.J., Humphries, S.E., Hales, C.N.

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Long-read single molecule real-time (SMRT) sequencing of GBA1 locus in Gaucher disease national cohort from Argentina reveals high frequency of complex allele underlying severe ske... von Drelichman, Guillermo I., Fernández Escobar, Nicolas, Soberon, Barbara C., Basack, Nora F., Frabasil, Joaquin, Schenone, Andrea B., Aguilar, Gabriel, Larroudé, Maria S., Knight, James R., Zhao, Dejian, Ruan, Jiapeng, Mistry, Pramod K.

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Molecular Diagnosis of Wilson Disease von Butler, Patrice, McIntyre, Neil, Mistry, Pramod K.

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Management of liver failure in a haemophilic patient co‐infected with human immunodeficiency and hepatitis C viruses von Nitu, I. C., Lee, C. A., Dhillon, A. P., Mistry, P. K.

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Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1 von Grabowski, Gregory A., Kacena, Katherine, Cole, J. Alexander, Hollak, Carla E.M., Zhang, Lin, Yee, John, Mistry, Pramod K., Zimran, Ari, Charrow, Joel, vom Dahl, Stephan

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