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

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

Nurix Therapeutics, Inc. stock slid 40% after promising NX-5948 results in CLL. NX-5948 shows efficacy in heavily pre-treated ...

No doubt 2025 will usher in many more advances in CRISPR therapies, thanks to the FDA’s new Platform Technology Designation ...

Rodriguez has filed a new measure that would allow patients suffering from severe illnesses to have individualized treatment options.

Using hormone replacement therapy (HRT) to reduce cardiovascular risk during menopause remains a topic of debate. The ...

Discover Stoke Therapeutics' innovative RNA-based treatments like zorevunersen for Dravet syndrome, with a promising pipeline ...

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...

Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million2025 Financial ...

Ultragenyx Pharmaceutical RARE reported fourth-quarter 2024 loss of $1.39 per share, wider than the Zacks Consensus Estimate ...