Less than a month after researchers unveiled that a “ticking DNA clock” is behind Huntington’s disease (HD), scientists have ...

The nonprofit foundation, n-Lorem developed personalized medicine for a child with rare leukodystrophy.

Both Tabrizi and Yang think that knocking down MSH3 could stave off HD pathology. Tabrizi noted that MSH3 also modifies other run-on repeats, including those causing fragile X syndrome, myotonic ...

An inhaled gene therapy for cystic fibrosis developed by Boehringer Ingelheim is due to start clinical testing shortly, hoping to become a first-in-class treatment for the genetic disorder.

Lung cancer is one of the leading causes of cancer mortality worldwide. Non-small cell lung cancer (NSCLC) accounts for about ...

Biogen and Stoke Therapeutics have announced a partnership aimed at advancing a disease-modifying treatment for Dravet ...

Biogen has entered a collaboration with Stoke Therapeutics to develop and and commercialise zorevunersen to treat Dravet ...

The partnership splits the rights to Stoke’s epilepsy antisense oligonucleotide, with up to $385 million in potential ...