Barth Syndrome Latest Advances

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.

Journal: Stem cell reviews and reports

Published: September 30, 2025

Barth syndrome: Natural history in infants and young children.

Journal: Molecular genetics and metabolism

Published: August 28, 2025

Barth Syndrome Latest Advances

Find the Latest Research About Barth Syndrome

Case Report: Deletion in the 5' untranslated region of TAFAZZIN in a boy with Barth syndrome.

Case Report: Deletion in the 5' untranslated region of TAFAZZIN in a boy with Barth syndrome.

Generation of a pluripotent embryonic stem cell TAFAZZIN hESC model (WAe009-A-3H) of Barth syndrome.

Generation of a pluripotent embryonic stem cell TAFAZZIN hESC model (WAe009-A-3H) of Barth syndrome.

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.

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.

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.

Letter to the Editor: Battle Won but War Lost for Barth Syndrome Patients.

Letter to the Editor: Battle Won but War Lost for Barth Syndrome Patients.

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.

Deficient Cardiolipin Remodelling Alters Muscle Fibre Composition and Neuromuscular Connectivity in Barth Syndrome.

Deficient Cardiolipin Remodelling Alters Muscle Fibre Composition and Neuromuscular Connectivity in Barth Syndrome.

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.

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.

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.

Barth syndrome: Natural history in infants and young children.

Barth syndrome: Natural history in infants and young children.