Barth Syndrome Latest Advances

Find the Latest Research About Barth Syndrome

Last Updated: 02/24/2026

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Found 304 publications

Deficient Cardiolipin Remodeling Alters Muscle Fiber Composition and Neuromuscular Connectivity in Barth Syndrome.

Deficient Cardiolipin Remodeling Alters Muscle Fiber Composition and Neuromuscular Connectivity in Barth Syndrome.

Journal: bioRxiv : the preprint server for biology
Published: November 24, 2025

Real-world disease burden and health care resource utilization for patients with Barth syndrome.

Real-world disease burden and health care resource utilization for patients with Barth syndrome.

Journal: Journal of medical economics
Published: November 21, 2025

Elamipretide: The first cardiolipin-directed mitochondrial therapeutic for Barth syndrome approved under accelerated approval.

Elamipretide: The first cardiolipin-directed mitochondrial therapeutic for Barth syndrome approved under accelerated approval.

Journal: Drug discoveries & therapeutics
Published: November 19, 2025

3D bioprinted myocardium patches for rare Barth syndrome: TAZ mutation correction in cardioblasts.

3D bioprinted myocardium patches for rare Barth syndrome: TAZ mutation correction in cardioblasts.

Journal: Annals of medicine and surgery (2012)
Published: November 09, 2025

Letter to the Editor: CRISPR-based gene editing for cardiac protection in Barth syndrome.

Letter to the Editor: CRISPR-based gene editing for cardiac protection in Barth syndrome.

Journal: Annals of medicine and surgery (2012)
Published: October 09, 2025

Pharmacological increases in circulating ketones fail to alleviate the hypertrophic cardiomyopathy present in the Tafazzin knockdown mouse model of Barth syndrome.

Pharmacological increases in circulating ketones fail to alleviate the hypertrophic cardiomyopathy present in the Tafazzin knockdown mouse model of Barth syndrome.

Journal: Journal of pharmacy & pharmaceutical sciences : a publication of the Canadian Society for Pharmaceutical Sciences, Societe canadienne des sciences pharmaceutiques
Published: October 01, 2025

Correction to: Granulopoietic Dysregulation in a Patient-Tailored Mouse Model of Barth Syndrome.

Correction to: Granulopoietic Dysregulation in a Patient-Tailored Mouse Model of Barth Syndrome.

Journal: Stem cell reviews and reports
Published: September 30, 2025

Barth syndrome: Natural history in infants and young children.

Barth syndrome: Natural history in infants and young children.

Journal: Molecular genetics and metabolism
Published: August 28, 2025

The B-lymphoblastoid model in Barth syndrome.

The B-lymphoblastoid model in Barth syndrome.

Journal: Biochimica et biophysica acta. Molecular and cell biology of lipids
Published: August 06, 2025

Granulopoietic Dysregulation in a Patient-Tailored Mouse Model of Barth Syndrome.

Granulopoietic Dysregulation in a Patient-Tailored Mouse Model of Barth Syndrome.

Journal: Stem cell reviews and reports
Published: July 11, 2025

A novel TAFAZZIN gene variant c.525_533del causing Barth syndrome and leading to heart transplantation: a case report.

A novel TAFAZZIN gene variant c.525_533del causing Barth syndrome and leading to heart transplantation: a case report.

Journal: Frontiers in pediatrics
Published: May 23, 2025

Elamipretide in the Management of Barth Syndrome: Current Evidence and a Case Report.

Elamipretide in the Management of Barth Syndrome: Current Evidence and a Case Report.

Journal: Molecular genetics and metabolism
Published: May 14, 2025
Showing 1-12 of 304

Last Updated: 02/24/2026