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Aberrant progranulin, YKL-40, cathepsin D and cathepsin S in Gaucher disease.
Afinogenova Y, Ruan J, Yang R, Kleytman N, Pastores G, Lischuk A, Mistry PK. Afinogenova Y, et al. Mol Genet Metab. 2019 Sep-Oct;128(1-2):62-67. doi: 10.1016/j.ymgme.2019.07.014. Epub 2019 Jul 23. Mol Genet Metab. 2019. PMID: 31358474 Free PMC article.
Enzyme replacement therapy resulted in a significant reversal of elevated cathepsin D and S but there was no change in progranulin and YKL-40 levels. Patients with persistent splenomegaly after long-term enzyme replacement therapy had significantly higher serum YKL-40 than …
Enzyme replacement therapy resulted in a significant reversal of elevated cathepsin D and S but there was no change in progranulin and YKL-4 …
Opening windows for bone remodeling through a SLIT.
Iqbal J, Yuen T, Kim SM, Zaidi M. Iqbal J, et al. J Clin Invest. 2018 Apr 2;128(4):1255-1257. doi: 10.1172/JCI120325. Epub 2018 Mar 5. J Clin Invest. 2018. PMID: 29504950 Free PMC article.
Bone-forming agents have been explored clinically to increase bone density; however, long-term efficacy of these strategies is limited due to the accompanying increase in resorption in response to increased bone formation. ...
Bone-forming agents have been explored clinically to increase bone density; however, long-term efficacy of these strategies is limite …
Outcomes after 18 months of eliglustat therapy in treatment-naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial.
Mistry PK, Lukina E, Ben Turkia H, Shankar SP, Baris H, Ghosn M, Mehta A, Packman S, Pastores G, Petakov M, Assouline S, Balwani M, Danda S, Hadjiev E, Ortega A, Gaemers SJM, Tayag R, Peterschmitt MJ. Mistry PK, et al. Am J Hematol. 2017 Nov;92(11):1170-1176. doi: 10.1002/ajh.24877. Epub 2017 Oct 3. Am J Hematol. 2017. PMID: 28762527 Free PMC article. Clinical Trial.
Eliglustat was well-tolerated, and there were no new safety concerns with longer-term exposure....
Eliglustat was well-tolerated, and there were no new safety concerns with longer-term exposure....
Clonal Immunoglobulin against Lysolipids in the Origin of Myeloma.
Nair S, Branagan AR, Liu J, Boddupalli CS, Mistry PK, Dhodapkar MV. Nair S, et al. N Engl J Med. 2016 Feb 11;374(6):555-61. doi: 10.1056/NEJMoa1508808. N Engl J Med. 2016. PMID: 26863356 Free PMC article.
Substrate reduction ameliorates Gaucher's disease-associated gammopathy in mice. Thus, long-term immune activation by lysolipids may underlie both Gaucher's disease-associated gammopathies and some sporadic monoclonal gammopathies....
Substrate reduction ameliorates Gaucher's disease-associated gammopathy in mice. Thus, long-term immune activation by lysolipids may …
Questioning the association between bisphosphonates and atypical femoral fractures.
Pazianas M, Kim SM, Yuen T, Sun L, Epstein S, Zaidi M. Pazianas M, et al. Ann N Y Acad Sci. 2015 Jan;1335(1):1-9. doi: 10.1111/nyas.12551. Epub 2014 Oct 7. Ann N Y Acad Sci. 2015. PMID: 25294742 Free PMC article. Review.
This question has prompted calls for a broader examination of the long-term effects of bisphosphonate use. An attempt by the Food and Drug Administration to garner consensus and provide definitive views was not successful. ...
This question has prompted calls for a broader examination of the long-term effects of bisphosphonate use. An attempt by the Food and …