Tag Archive for: Orphan diseases

The Fragile X mutation is a type of genetic mutation called a repeat expansion. This repeat expansion occurs on the FMR1 gene, on the long arm of the X chromosome leading to gene silencing if more than 200 repeats occur.FRAXA Ewsearch Organisation
Partnership

Servier acquires Kaerus Bioscience’s FXS candidate for US$450m

In a US$450m deal with UK-based Kaerus Bioscience Ltd, Servier SA has acquired the development and commercialisation rights to KER-0193, a Phase II-ready drug candidate for the treatment of rare Fragile X syndrome, the most common monogenic cause of autism.

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© Olga Yastremska, New Africa, freepik
Orphan diseases

Breakthrough in microtia treatment

Researchers developed an innovative ear transplant method to treat microtia using tissue-engineered human skin and bioprinted cartilage.

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