All Barth syndrome articles
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NewsPotential 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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ArticleThe 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 ...