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Treatment of Barth Syndrome by Cardiolipin Manipulation ...


Treatment of Barth Syndrome by Cardiolipin Manipulation ... - PubMed

The aim of this study is to determine whether bezafibrate (and resveratrol in vitro) will increase mitochondrial biogenesis and potentially modify the cellular ...

Treatment of Barth Syndrome by Cardiolipin Manipulation ... - NCBI

The PPAR pan-agonist bezafibrate ameliorates cardiomyopathy in a mouse model of Barth syndrome. Orphanet J Rare Dis. 2017 Mar 09;12(1):49.

Treatment of Barth Syndrome by Cardiolipin Manipulation ...

Background: Barth syndrome is a rare, life-threatening, X-linked recessive genetic disease that predominantly affects young males and is ...

CARDIOMAN Clinical Trial for Barth Syndrome

Treatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN): A randomised placebo controlled pilot trial conducted by the nationally commissioned ...

Award 12/205/56

Award 12/205/56. SolrDocument{id=12/205/56, award_type=Research, award_title=Treatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN): A ...

A randomised placebo controlled pilot trial conducted by ... - Orphanet

Treatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN): A randomised placebo controlled pilot trial conducted by the nationally commissioned Barth ...

(PDF) Treatment of Barth Syndrome by Cardiolipin Manipulation ...

Background Barth syndrome is a rare, life-threatening X-linked recessive mitochondrial disorder of lipid metabolism primarily affecting males.

Treatment of Barth Syndrome by Cardiolipin Manipulation ...

Currently, there is no definitive treatment for Barth syndrome other than interventions to ameliorate acute symptoms, such as heart failure, ...

Treatment of Barth Syndrome by - HTA Database - inahta

Treatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN): a randomised placebo-controlled pilot trial conducted by the nationally commissioned Barth ...

Current and future treatment approaches for Barth syndrome

Management of the infant with severe heart failure due to Barth syndrome may require use of intravenous inotropes and diuretics for ...

Investigating bezafibrate as a treatment for Barth syndrome

Therefore, the use of bezafibrate in this clinical trial is the first potential treatment available for the alleviation of the abnormal cardiolipin ratio ...

The heart of the matter: Elamipretide in treating Barth syndrome

Elamipretide was the first drug with the potential to treat mitochondria in heart failure and by extension in Barth syndrome.

CARDIOMAN - Health Research Authority

Research type. Research Study · Full title. Treatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN): A randomised placebo controlled pilot trial ...

New targets for monitoring and therapy in Barth syndrome - Nature

Barth syndrome (BTHS), an X-linked disorder caused by defects in TAZ, is the only known single-gene disorder of cardiolipin remodeling.

Barth syndrome: A life-threatening disorder caused by abnormal ...

Commonly, BTHS patients require cardiac transplantation29. The combination of prophylactic antibiotics and G-CSF are used to treat symptomatic neutropenia. To ...

Treatment of Barth Syndrome by Cardiolipin Manipulation ...

Treatment of Barth Syndrome by Cardiolipin Manipulation (CARDIOMAN) With Bezafibrate: Protocol for a Randomized Placebo-Controlled Pilot Trial Conducted in the ...

Treatment of Barth Syndrome by CARDIOlipin MANipulation ...

Treatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN): a randomised placebo-controlled pilot trial conducted by the nationally ...

Clinical Trials : Clinical Trials : Research - Barth Syndrome Foundation

... cure for Barth syndrome. A fundamental ... For more information please click here. Treatment of Barth Syndrome by CARDIOlipin MANipulation ...

Cardiolipin remodeling in Barth syndrome and other hereditary ...

Specifically, AAV-TAZ treatment improved mitochondrial morphology, ATP production and ROS emission in patient-derived fibroblasts [225], and ameliorated cardiac ...

A phase 2/3 randomized clinical trial followed by an open-label ...

To evaluate effectiveness of elamipretide in Barth syndrome (BTHS), a genetic condition of defects in TAZ, which causes abnormal cardiolipin on the inner ...