Summarized by Plex Health
Last Updated: 02 May 2022
skeletal improvement in patients with gaucher disease type 1: a phase 2 trial of oral eliglustat. "skeletal improvement in patients with gaucher disease type 1: a phase 2 trial of oral eliglustat.", by Kamath RS, Lukina E, Watman N, Dragosky M, Pastores GM, Arreguin EA, Rosenbaum H, Zimran A, Aguzzi R, Puga AC, Norfleet AM, Peterschmitt MJ, Rosenthal DI. Fig2: Dark marrow improvement in two patients after 4 years of eliglustat therapy. Top Coronal T1-weighted MR images of the proximal right femur of a 55-year-old female patient at baseline (a) and at 48 months...

Eliglustat minimizes the development of a particular protein in the body in people with type 1 Gaucher disease. Gaucher disease is a hereditary problem in which the body lacks the enzyme required to break down particular fatty materials. Eliglustat is used to treat mild to modest type 1 Gaucher disease in adults. If a specific liver enzyme in your body breaks down or metabolizes drugs at a specific rate, eliglustat is used only. Eliglustat may enhance the problem of the liver, spleen, bones, and blood cells in people with Type I Gaucher disease. Eliglustat may be used for purposes not noted in this medication overview. Eliglustat can cause a major heart problem, particularly if you use specific other medicines at the same time. Tell your medical professional about all medications you use. Some drugs can increase or lower your blood levels of eliglustat, which may cause side effects or make eliglustat less efficient. Eliglustat can also impact blood levels of particular other drugs, making them less effective or increasing side effects. It is not recognized whether eliglustat will hurt an expected infant. Your doctor will carry out a genotype blood test to make sure eliglustat is the right therapy for you. Your physician might periodically change your dosage. Wait at least 24 hours after your last dosage of the other medicine prior to you start taking eliglustat if you switched over to eliglustat from another enzyme substitute medicine. Eliglustat can cause a severe heart trouble. Take the medication as soon as you can, however avoid the missed dose if it is almost time for your following dosage.

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Research Papers' Summaries

PubChem - CovalentUnitCount

(Table source)
23652731C23H36N2O4404.5CCCCCCCC(=O)NC(CN1CCCC1)C(C2=CC3=C(C=C2)OCCO3)OCCCCCCCC(=O)N[[email protected]](CN1CCCC1)[[email protected]@H](C2=CC3=C(C=C2)OCCO3)OInChI=1S/C23H36N2O4/c1-2-3-4-5-6-9-22(26)24-19(17-25-12-7-8-13-25)23(27)18-10-11-20-21(16-18)29-15-14-28-20/h10-11,16,19,23,27H,2-9,12-15,17H2,1H3,(H,24,26)/t19-,23-/m1/s1FJZZPCZKBUKGGU-AUSIDOKSSA-NN-[(1R,2R)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-pyrrolidin-1-ylpropan-2-yl]octanamide3.7404.26750763404.2675076371484025112902200001
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