Researchers from the Huntington's Disease Center, UCL Queen Square Institute of Neurology, and UK DRI at UCL have taken major ...

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.

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 ...

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

Ionis relies on upfront payments and licensing fees from partners, as well as Spinraza royalties, to drive revenue today, but its rapidly advancing portfolio should help diversify revenue. 2025 is ...

A new discovery offers hope for Huntington’s disease. This discovery provides hope that a DNA repair process may help slow or ...

Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease characterized by the progressive degeneration ...

Market OverviewThe Global RNA Therapeutics Market is valued at USD 13.3 Billion in 2023 and is projected to reach a value ...

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

The deal, which features up to $385 million in milestones, gives Biogen exclusive rights to commercialize Stoke’s ...

With its recent data drop for an oligonucleotide candidate, Dyne Therapeutics signals it may become a frontrunner in this ...

Biogen Inc. (NASDAQ:BIIB) and Stoke Therapeutics, Inc. (NASDAQ:STOK) collaborated to develop and commercialize zorevunersen ...