The FDA has extended the review period for the New Drug Application for elamipretide for the treatment of Barth syndrome.

Baylen Dupree, 22, stars in a new TLC series after she built a following on social media. She wants people to know what it's ...

how to correct it", says Barth. "Neurogenerative diseases for example can be the result of misregulated genes during early stages of pregnancy. In fact, there are a couple of severe diseases, such as ...

A new study finds that female individuals are less likely to be diagnosed with the syndrome, take longer to receive a diagnosis and are older when they are diagnosed than male individuals.

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Rett syndrome is a severe and complex neurodevelopmental disorder. Trofinetide, a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1 or IGF-1, is already approved in the ...

Quoin Pharmaceuticals (QNRX) announces interim clinical data from its ongoing Investigator Pediatric Netherton Syndrome clinical study. The Investigator Pediatric Study continues to demonstrate ...

Last month, media reports claimed that the accelerated accumulation of space debris had raised the real possibility of a "collision cascading" effect called the Kessler Syndrome. Kessler Syndrome ...