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Potential of small molecule therapeutics for Barth syndrome
Novel findings about the tafazzin gene offers a potential new target and drug candidate for Barth syndrome.
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Barth syndrome
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The genetic modifier approach: identifying the right target for rare diseases
In this article, Dr Vincent Blomen, Senior Director of Target Discovery at Scenic Biotech, takes us through the realm of rare diseases. In the case of most of these diseases, a singular genetic anomaly often reigns supreme, yet its impact on patients can be vastly divergent. Enter modifier genes, the…
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TAZ gene therapy reverses Barth syndrome in mice
Researchers have successfully treated cardiac dysfuntion in mice models of Barth syndrome by using a gene therapy to replace TAZ.
