The field of gene editing recently got a major boost from Massachusetts-based Beam Therapeutics, and the company’s Durham ...

Over 95% of babies now diagnosed with SMA are treated with Zolgensma gene therapy, per Daniel Grant, vice president for ...

The award recognizes Esteve Química as a top-performing contract development and manufacturing organization (CDMO) for its excellence in pharmaceutical development and manufacturing services. It also ...

The UK has long been a destination for global businesses, offering a dynamic environment that fosters innovation and ...

Phase 1 clinical trial of CMP-CPS-001 in Urea Cycle Disorders (UCDs) ongoing, with dosing completed in two of four multiple ascending dose ...

Michelle Harlan, who has sickle beta thalassemia, wants to help others by getting a doctoral degree in clinical psychology.

Long-term treatment with Duvyzat (givinostat) may help delay the loss of walking ability and lessen the decline in lung ...