Page last updated: 2024-10-19

phosphorylcholine and Niemann-Pick Disease, Type A

phosphorylcholine has been researched along with Niemann-Pick Disease, Type A in 3 studies

Phosphorylcholine: Calcium and magnesium salts used therapeutically in hepatobiliary dysfunction.
phosphocholine : The phosphate of choline; and the parent compound of the phosphocholine family.

Niemann-Pick Disease, Type A: The classic infantile form of Niemann-Pick Disease, caused by mutation in SPHINGOMYELIN PHOSPHODIESTERASE. It is characterized by accumulation of SPHINGOMYELINS in the cells of the MONONUCLEAR PHAGOCYTE SYSTEM and other cell throughout the body leading to cell death. Clinical signs include JAUNDICE, hepatosplenomegaly, and severe brain damage.

Research Excerpts

ExcerptRelevanceReference
" The majority of adverse events were mild and included headache, nausea, and abdominal pain."2.87Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months. ( Diaz, GA; Ji, AJ; Jouvin, MH; Lachmann, RH; Nandy, I; Puga, AC; Wasserstein, MP, 2018)
"Acid sphingomyelinase deficiency (ASMd, Niemann-Pick disease A/B) and Niemann-Pick type C disease (NPC) share core clinical symptoms."1.46Quantitation of plasmatic lysosphingomyelin and lysosphingomyelin-509 for differential screening of Niemann-Pick A/B and C diseases. ( Asfaw, B; Gulinello, ME; Jahnova, H; Kuchar, L; Ledvinova, J; Lugowska, A; Malinova, V; Poupetova, H; Sikora, J, 2017)

Research

Studies (3)

TimeframeStudies, this research(%)All Research%
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's1 (33.33)29.6817
2010's2 (66.67)24.3611
2020's0 (0.00)2.80

Authors

AuthorsStudies
Wasserstein, MP1
Diaz, GA1
Lachmann, RH1
Jouvin, MH1
Nandy, I1
Ji, AJ1
Puga, AC1
Kuchar, L1
Sikora, J1
Gulinello, ME1
Poupetova, H1
Lugowska, A1
Malinova, V1
Jahnova, H1
Asfaw, B1
Ledvinova, J1
Chiulli, N1
Codazzi, F1
Di Cesare, A1
Gravaghi, C1
Zacchetti, D1
Grohovaz, F1

Clinical Trials (1)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
A Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency[NCT02004704]Phase 225 participants (Actual)Interventional2013-12-04Completed
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trials

1 trial available for phosphorylcholine and Niemann-Pick Disease, Type A

ArticleYear
Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months.
    Journal of inherited metabolic disease, 2018, Volume: 41, Issue:5

    Topics: Adult; Biomarkers; Bone Density; Enzyme Replacement Therapy; Female; Hexosaminidases; Humans; Lipids

2018

Other Studies

2 other studies available for phosphorylcholine and Niemann-Pick Disease, Type A

ArticleYear
Quantitation of plasmatic lysosphingomyelin and lysosphingomyelin-509 for differential screening of Niemann-Pick A/B and C diseases.
    Analytical biochemistry, 2017, May-15, Volume: 525

    Topics: Biomarkers; Case-Control Studies; Chromatography, Liquid; Dried Blood Spot Testing; Humans; Niemann-

2017
Sphingosylphosphocholine effects on cultured astrocytes reveal mechanisms potentially involved in neurotoxicity in Niemann-Pick type A disease.
    The European journal of neuroscience, 2007, Volume: 26, Issue:4

    Topics: Animals; Apoptosis; Astrocytes; Cell Proliferation; Cells, Cultured; Cerebral Cortex; Fluorescent Dy

2007