Barth Syndrome Latest Advances

Omega-3 fatty acid supplementation improves skeletal muscle mitochondrial function in a model of Barth syndrome.

Journal: JCI insight

Published: June 09, 2026

Does the beta subunit of mitochondrial trifunctional protein modulate monolysocardiolipin acylation to cardiolipin?

Journal: Biochemistry and cell biology = Biochimie et biologie cellulaire

Published: June 05, 2026

Saturated cardiolipins are potent disruptors of inner mitochondrial membrane structure and function.

Journal: bioRxiv : the preprint server for biology

Published: April 27, 2026

Is an emerging pharmacotherapeutic era for rare mitochondrial diseases here?

Journal: Cell metabolism

Published: April 18, 2026

From Pharmacodynamic Biomarker to Evaluating Treatment Response: Biomarkers in Primary Mitochondrial Diseases.

Journal: Clinical and translational science

Published: March 13, 2026

Insights into Neutrophil Dysfunction in Inherited Metabolic Disorders.

Journal: Journal of innate immunity

Published: February 26, 2026

2025 FDA TIDES (Peptides and Oligonucleotides) Harvest.

Journal: Pharmaceuticals (Basel, Switzerland)

Published: January 12, 2026

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

Journal: Frontiers in cardiovascular medicine

Published: December 11, 2025

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

Journal: Stem cell research

Published: December 11, 2025

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.

Journal: Journal of medical economics

Published: November 21, 2025

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

Journal: Drug discoveries & therapeutics

Published: November 19, 2025

Barth Syndrome Latest Advances

Find the Latest Research About Barth Syndrome

Omega-3 fatty acid supplementation improves skeletal muscle mitochondrial function in a model of Barth syndrome.

Omega-3 fatty acid supplementation improves skeletal muscle mitochondrial function in a model of Barth syndrome.

Does the beta subunit of mitochondrial trifunctional protein modulate monolysocardiolipin acylation to cardiolipin?

Does the beta subunit of mitochondrial trifunctional protein modulate monolysocardiolipin acylation to cardiolipin?

Saturated cardiolipins are potent disruptors of inner mitochondrial membrane structure and function.

Saturated cardiolipins are potent disruptors of inner mitochondrial membrane structure and function.

Is an emerging pharmacotherapeutic era for rare mitochondrial diseases here?

Is an emerging pharmacotherapeutic era for rare mitochondrial diseases here?

From Pharmacodynamic Biomarker to Evaluating Treatment Response: Biomarkers in Primary Mitochondrial Diseases.

From Pharmacodynamic Biomarker to Evaluating Treatment Response: Biomarkers in Primary Mitochondrial Diseases.

Insights into Neutrophil Dysfunction in Inherited Metabolic Disorders.

Insights into Neutrophil Dysfunction in Inherited Metabolic Disorders.

2025 FDA TIDES (Peptides and Oligonucleotides) Harvest.

2025 FDA TIDES (Peptides and Oligonucleotides) Harvest.

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.