Cargando…

Dietary management and major clinical events in patients with longchain fatty acid oxidation disorders enrolled in a phase 2 triheptanoin study

BACKGROUND & AIMS: Long-chain fatty acid oxidation disorders (LC-FAOD) are rare, life-threatening, autosomal recessive disorders that lead to energy depletion and major clinical events (MCEs), such as acute metabolic crises of hypoglycemia, cardiomyopathy, and rhabdomyolysis. The aim of this stu...

Descripción completa

Detalles Bibliográficos
Autores principales:	Vockley, Jerry, Longo, Nicola, Madden, Megan, Dwyer, Lauren, Mu, Yunming, Chen, Chao-Yin, Cataldo, Jason
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8567087/ https://www.ncbi.nlm.nih.gov/pubmed/33487279 http://dx.doi.org/10.1016/j.clnesp.2020.11.018

Ejemplares similares

Effects of triheptanoin (UX007) in patients with long‐chain fatty acid oxidation disorders: Results from an open‐label, long‐term extension study
por: Vockley, Jerry, et al.
Publicado: (2020)

Physiological Perspectives on the Use of Triheptanoin as Anaplerotic Therapy for Long Chain Fatty Acid Oxidation Disorders
por: Sklirou, Evgenia, et al.
Publicado: (2021)

Triheptanoin: First Approval
por: Shirley, Matt
Publicado: (2020)

Correction to: Triheptanoin: First Approval
por: Shirley, Matt
Publicado: (2020)

No effect of triheptanoin on exercise performance in McArdle disease
por: Madsen, Karen L., et al.
Publicado: (2019)

Long-term experience with triheptanoin in 12 Austrian patients with long-chain fatty acid oxidation disorders
por: Zöggeler, Thomas, et al.
Publicado: (2021)

The Pharmacokinetics of Triheptanoin and Its Metabolites in Healthy Subjects and Patients With Long‐Chain Fatty Acid Oxidation Disorders
por: Lee, Sun Ku, et al.
Publicado: (2021)

Population Pharmacokinetics of Heptanoate in Healthy Subjects and Patients With Long‐Chain Fatty Acid Oxidation Disorders Treated With Triheptanoin
por: Lee, Sun Ku, et al.
Publicado: (2022)

Triheptanoin dramatically reduces paroxysmal motor disorder in patients with GLUT1 deficiency
por: Mochel, Fanny, et al.
Publicado: (2016)

Exploring triheptanoin as treatment for short chain enoyl CoA hydratase deficiency
por: Engelstad, Kristin, et al.
Publicado: (2021)

A randomized, controlled, double-blind, crossover trial of triheptanoin in alternating hemiplegia of childhood
por: Hainque, Elodie, et al.
Publicado: (2017)

Randomized trial of add‐on triheptanoin vs medium chain triglycerides in adults with refractory epilepsy
por: Borges, Karin, et al.
Publicado: (2019)

Open‐label long‐term treatment of add‐on triheptanoin in adults with drug‐resistant epilepsy
por: Borges, Karin, et al.
Publicado: (2020)

Triheptanoin – Novel therapeutic approach for the ultra-rare disease mitochondrial malate dehydrogenase deficiency
por: Laemmle, Alexander, et al.
Publicado: (2021)

Triheptanoin Protects Motor Neurons and Delays the Onset of Motor Symptoms in a Mouse Model of Amyotrophic Lateral Sclerosis
por: Tefera, Tesfaye W., et al.
Publicado: (2016)

Combination of triheptanoin with the ketogenic diet in Glucose transporter type 1 deficiency (G1D)
por: Avila, Adrian, et al.
Publicado: (2023)

A randomized, double‐blind trial of triheptanoin for drug‐resistant epilepsy in glucose transporter 1 deficiency syndrome
por: Striano, Pasquale, et al.
Publicado: (2022)

Biomarkers for drug development in propionic and methylmalonic acidemias
por: Longo, Nicola, et al.
Publicado: (2022)

Maximum dose, safety, tolerability and ketonemia after triheptanoin in glucose transporter type 1 deficiency (G1D)
por: Málaga, Ignacio, et al.
Publicado: (2023)

Long-term follow-up in an open-label trial of triheptanoin in GLUT1 deficiency syndrome: a sustained dramatic effect
por: Hainque, Elodie, et al.
Publicado: (2019)

Impact of newborn screening on the reported incidence and clinical outcomes associated with medium- and long-chain fatty acid oxidation disorders
por: Marsden, Deborah, et al.
Publicado: (2021)

Outcomes and genotype correlations in patients with mitochondrial trifunctional protein or isolated long chain 3-hydroxyacyl-CoA dehydrogenase deficiency enrolled in the IBEM-IS database
por: Lim, Chelsey Chaehee, et al.
Publicado: (2022)

Improving diagnosis of mitochondrial fatty-acid oxidation disorders
por: Vianey-Saban, Christine, et al.
Publicado: (2023)

Effects of Triheptanoin on Mitochondrial Respiration and Glycolysis in Cultured Fibroblasts from Neutral Lipid Storage Disease Type M (NLSD-M) Patients
por: Noguera, Nelida Inés, et al.
Publicado: (2023)

Anaplerotic Triheptanoin Diet Enhances Mitochondrial Substrate Use to Remodel the Metabolome and Improve Lifespan, Motor Function, and Sociability in MeCP2-Null Mice
por: Park, Min Jung, et al.
Publicado: (2014)

Evaluation of Preconception Dietary Patterns in Women Enrolled in a Multisite Study
por: Lim, Shan Xuan, et al.
Publicado: (2022)

Contraceptive Provision to Women With Intellectual and Developmental Disabilities Enrolled in Medicaid
por: Mitchell, Lauren, et al.
Publicado: (2023)

Dietary supplement use among health care professionals enrolled in an online curriculum on herbs and dietary supplements
por: Gardiner, Paula, et al.
Publicado: (2006)

Mitochondrial morphology, bioenergetics and proteomic responses in fatty acid oxidation disorders
por: Raimo, Serena, et al.
Publicado: (2021)

Lipidomic and Proteomic Alterations Induced by Even and Odd Medium-Chain Fatty Acids on Fibroblasts of Long-Chain Fatty Acid Oxidation Disorders
por: Alatibi, Khaled I., et al.
Publicado: (2021)

COVID-19 trial co-enrolment and subsequent enrolment
por: Gertner, Elie
Publicado: (2020)

Depression and anxiety in cancer patient enrolled in clinical trials with serious adverse events
por: Peng, Zhen, et al.
Publicado: (2023)

Different Lipid Signature in Fibroblasts of Long-Chain Fatty Acid Oxidation Disorders
por: Alatibi, Khaled I., et al.
Publicado: (2021)

Changes in use of herbs and dietary supplements (HDS) among clinicians enrolled in an online curriculum
por: Kemper, Kathi J, et al.
Publicado: (2007)

Dietary Habits of Students Enrolled in Faculties of Health Sciences: A Cross-sectional Study
por: AlJohani, Sarah, et al.
Publicado: (2019)

Risk Differential Between Medicare Beneficiaries Enrolled and Not Enrolled in an HMO
por: Eggers, Paul
Publicado: (1980)

Traumatic childhood events of parents enrolled in the Avon Longitudinal Study of Parents and Children (ALSPAC)
por: Ellis, Genette, et al.
Publicado: (2020)

Childhood life events of women enrolled in the Avon Longitudinal Study of Parents & Children (ALSPAC)
por: Gregory, Steven, et al.
Publicado: (2023)

Dietary Quality Maintained among Overweight Brazilian Women Enrolled in a Primary Healthcare Service
por: Martins Horta, Paula, et al.
Publicado: (2014)

Barriers to enrolment into a professional upgrading programme for enrolled nurses in Kenya
por: Lakati, Alice, et al.
Publicado: (2012)

Cannot write session to /tmp/vufind_sessions/sess_rsogndteopt0hqbtq4mgdpm83n