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Found 9 clinical trials
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190

throughout the body and is responsible for the breakdown of glycosphingolipids, deficiency of which results in the accumulation of specific glycosphingolipids that are associated with the pathophysiology of

deficiency
deficit
gene therapy
glycosphingolipids
  • 17 views
  • 25 Jan, 2021
GSL Synthetase Inhibitor or in Combination With Immune Checkpoint Inhibitor in Previously Treated Blood and Solid Tumor

inhibit glycosphingolipids synthesis and restore HLA-I antigen presentation and transform the immunogenicity of tumor cells. Therefore,GSL synthetase inhibitor eliglustat monotherapy or in combination

solid tumor
cancer
  • 0 views
  • 14 May, 2022
  • 1 location
Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) (EMTISTD)

disease. Zavesca passes blood brain barrier, so causes reduction of cholesterol and glycosphingolipids CNS neurons and relief of neurologic manifestations. Improvements were seen in oculomotor function

  • 36 views
  • 19 Apr, 2022
  • 3 locations
Role of Adiposomes in Endothelial Dysfunction

endothelial cells in OB-T2D subjects. On top of these lipid species that the investigators propose to be carried by adiposomes are glycosphingolipids (GSLs). These lipids originate from the glycosylation of

  • 0 views
  • 24 May, 2022
  • 1 location
Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 (LEAP2MONO)  

This is a parallel arm, Phase 3, double-blind, double-dummy, active-comparator, 2 arm study to evaluate the efficacy and safety of daily oral venglustat versus intravenous Cerezyme infusions every two weeks for improvement or stabilization of the neurological manifestations and maintenance of systemic disease stability in participants aged ≥12 and <18 …

neuronopathic gaucher disease
bone pain
cerezyme
deficiency
alglucerase
  • 6 views
  • 16 Jun, 2022
  • 1 location
Evaluating the Association Between Sphingolipid Metabolites and Post-hepatectomy Liver Failure

include ceramides (CER), sphingomyelins (SM), glycosphingolipids (GSL), sphingosine (SPH), and sphingosine-1-phosphate (S1P) are multi-functional lipids that regulates cell proliferation, cell survival

  • 0 views
  • 22 Jan, 2021
  • 1 location
Epidemiological Study of Fabry Disease Screening in Chronic Kidney Disease Patients

glycosphingolipids, predominantly globotriaosylceramide (Gb3), within cell lysosomes throughout the body. In patients at the second or third decade, progressive proteinuria, decline in glomerular filtration

lyso-gb3
gla gene
globotriaosylceramide
deficiency
deficit
  • 0 views
  • 29 Sep, 2021
  • 1 location
Trehalose Administration in Subjects With Spastic Paraplegia 11 (3AL-SPG11) (3AL-SPG11)

Hereditary spastic paraparesis type 11 (SPG11) is caused by mutations in the SPG11 gene that produces spatacsin, a protein involved in lysosomal function.

  • 0 views
  • 26 Apr, 2022
  • 1 location
Natural History of Glycosphingolipid and Glycoprotein Lysosomal Storage Disorders

This study will evaluate children with glycosphingolipid (GSL) storage disorders to investigate brain changes that cause nervous system degeneration. No experimental treatments are offered in this study; participants will receive standard medical care for their disease. The information from this study may help researchers develop new therapies for these disorders …

MRI
deficiency
  • 76 views
  • 17 Jun, 2022
  • 1 location