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

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

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.

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 ...