Cargando…

Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis

BACKGROUND: Niemann-Pick Disease Type C (NPC) is an inherited, often fatal neurovisceral lysosomal storage disease characterized by cholesterol accumulation in every cell with few known treatments. Defects in cholesterol transport cause sequestration of unesterified cholesterol within the endolysoso...

Descripción completa

Detalles Bibliográficos
Autores principales:	Hastings, Caroline, Vieira, Camilo, Liu, Benny, Bascon, Cyrus, Gao, Claire, Wang, Raymond Y., Casey, Alicia, Hrynkow, Sharon
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2019
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6805667/ https://www.ncbi.nlm.nih.gov/pubmed/31639011 http://dx.doi.org/10.1186/s13023-019-1207-1

Ejemplares similares

Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick Disease type C1
por: Yergey, Alfred L., et al.
Publicado: (2017)

Correction: Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick Disease type C1
por: Yergey, Alfred L., et al.
Publicado: (2018)

Effects of intracerebroventricular administration of 2-hydroxypropyl-β-cyclodextrin in a patient with Niemann–Pick Type C disease
por: Matsuo, Muneaki, et al.
Publicado: (2014)

2-Hydroxypropyl-β-cyclodextrins and the Blood-Brain Barrier: Considerations for Niemann-Pick Disease Type C1
por: Calias, Pericles
Publicado: (2017)

The Effects of Combined Therapy With Metformin and Hydroxypropyl-β-Cyclodextrin in a Mouse Model of Niemann-Pick Disease Type C1
por: Du, Jiang, et al.
Publicado: (2022)

Long-term administration of intravenous Trappsol® Cyclo™ (HP-β-CD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II trial
por: Sharma, Reena, et al.
Publicado: (2023)

A phase 1/2 open label nonrandomized clinical trial of intravenous 2-hydroxypropyl-β-cyclodextrin for acute liver disease in infants with Niemann-Pick C1
por: Reynolds, Margaret, et al.
Publicado: (2021)

Efficacy and ototoxicity of different cyclodextrins in Niemann–Pick C disease
por: Davidson, Cristin D., et al.
Publicado: (2016)

Visual evoked potentials of Niemann-Pick type C1 mice reveal an impairment of the visual pathway that is rescued by 2-hydroxypropyl-ß-cyclodextrin
por: Palladino, Giampiero, et al.
Publicado: (2015)

Influence of Npc1 genotype on the toxicity of hydroxypropyl-β-cyclodextrin, a potentially therapeutic agent, in Niemann–Pick Type C disease models()
por: Tanaka, Yuta, et al.
Publicado: (2014)

A therapy with miglustat, 2-hydroxypropyl-ß-cyclodextrin and allopregnanolone restores splenic cholesterol homeostasis in Niemann-pick disease type C1
por: Neßlauer, Anna-Maria, et al.
Publicado: (2019)

Hydroxypropyl-beta and -gamma cyclodextrins rescue cholesterol accumulation in Niemann–Pick C1 mutant cell via lysosome-associated membrane protein 1
por: Singhal, Ashutosh, et al.
Publicado: (2018)

Pluronic based β-cyclodextrin polyrotaxanes for treatment of Niemann-Pick Type C disease
por: Collins, Christopher J., et al.
Publicado: (2017)

Intracerebroventricular Treatment with 2-Hydroxypropyl-β-Cyclodextrin Decreased Cerebellar and Hepatic Glycoprotein Nonmetastatic Melanoma Protein B (GPNMB) Expression in Niemann–Pick Disease Type C Model Mice
por: Fukaura, Madoka, et al.
Publicado: (2021)

Linear Cyclodextrin Polymer Prodrugs as Novel Therapeutics for Niemann-Pick Type C1 Disorder
por: Kulkarni, Aditya, et al.
Publicado: (2018)

Different solubilizing ability of cyclodextrin derivatives for cholesterol in Niemann–Pick disease type C treatment
por: Yamada, Yusei, et al.
Publicado: (2023)

Vascular endothelial growth factor improves the therapeutic effects of cyclodextrin in Niemann-Pick type C mice
por: Jeong, Min Seock, et al.
Publicado: (2019)

Niemann-Pick disease type C
por: Vanier, Marie T
Publicado: (2010)

Stem Cells and Niemann Pick Disease
por: Andolina, Marino
Publicado: (2014)

Stem Cells in Niemann-Pick Disease
por: Kim, Sun-Jung, et al.
Publicado: (2008)

Niemann-Pick disease and platelet dysfunction
Publicado: (2012)

Impact of Intravenous Trehalose Administration in Patients with Niemann–Pick Disease Types A and B
por: Mobini, Moein, et al.
Publicado: (2022)

Lysosomal and Mitochondrial Liaisons in Niemann-Pick Disease
por: Torres, Sandra, et al.
Publicado: (2017)

Niemann-Pick Disease: An Approach for Diagnosis in Adulthood
por: Patiño-Escobar, Bonell, et al.
Publicado: (2019)

Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression
por: Davidson, Cristin D., et al.
Publicado: (2009)

Inclusion Complex of Clomiphene Citrate with Hydroxypropyl-β-Cyclodextrin for Intravenous Injection: Formulation and Stability Studies
por: Annaji, Manjusha, et al.
Publicado: (2023)

The expanding boundaries of sphingolipid lysosomal storage diseases; insights from Niemann–Pick disease type C
por: Platt, Frances M.
Publicado: (2023)

The prognostic value of Niemann-Pick C1-like protein 1 and Niemann-Pick disease type C2 in hepatocellular carcinoma
por: Chen, Ke-Ji, et al.
Publicado: (2018)

Clinical disease characteristics of patients with Niemann-Pick Disease Type C: findings from the International Niemann-Pick Disease Registry (INPDR)
por: Bolton, Shaun C., et al.
Publicado: (2022)

In vivo Efficacy and Safety Evaluation of Lactosyl-β-cyclodextrin as a Therapeutic Agent for Hepatomegaly in Niemann-Pick Type C Disease
por: Maeda, Yuki, et al.
Publicado: (2019)

Methyl-β-cyclodextrin restores impaired autophagy flux in Niemann-Pick C1-deficient cells through activation of AMPK
por: Dai, Sheng, et al.
Publicado: (2017)

Clear Cell Chondrosarcoma in Association With Niemann-Pick Disease
por: Srikanth, K. N., et al.
Publicado: (2005)

Immune dysfunction in Niemann‐Pick disease type C
por: Platt, Nick, et al.
Publicado: (2015)

Anesthetic Management in a Child With Niemann-Pick Disease
por: Espahbodi, Ebrahim, et al.
Publicado: (2016)

Niemann-Pick Disease: An Underdiagnosed Lysosomal Storage Disorder
por: Panigrahi, Inusha, et al.
Publicado: (2019)

A calcium message for Niemann-Pick type C
por: Cologna, Stephanie M.
Publicado: (2019)

Treatment trials in Niemann-Pick type C disease
por: Sitarska, Dominika, et al.
Publicado: (2021)

Niemann-Pick type C proteins promote microautophagy by expanding raft-like membrane domains in the yeast vacuole
por: Tsuji, Takuma, et al.
Publicado: (2017)

Encapsulation Mechanism of Oxyresveratrol by β-Cyclodextrin and Hydroxypropyl-β-Cyclodextrin and Computational Analysis
por: He, Jianfei, et al.
Publicado: (2017)

Health insurance literacy and health services access barriers in Niemann–Pick disease: the patient and caregiver voice
por: Diaz, George A., et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_kii1bu9qc2o8tqasfv9kf6hpo0