The study will compare neladalkib to Genentech's ALK inhibitor Alecensa in patients who've had no prior systemic treatment.

The FDA requested Sarepta voluntarily stop distributing Elevidys after the death of a third patient who received one of the firm's gene therapies.

The privately funded basket trial makes good on its aim to enroll underrepresented groups amid a DEI-hostile federal funding ...

In a final draft guidance, the committee recommended the treatment for HR-positive HER2-negative early breast cancer patients, regardless of lymph node involvement.

Company touted Kisqali's "replacement power" as another blockbuster faces generic competition and expressed optimism about ...

For $1.8 million upfront and additional contingent and milestone payments, I-Mab gains full rights to givastomig's parental antibody.

NEW YORK – Sarepta Therapeutics on Wednesday said it would pivot to focus its rare genetic disease pipeline on siRNA drugs as part of a strategic restructuring plan to prioritize "high-value, high ...

A 51-year-old man with limb-girdle muscular dystrophy died after receiving Sarepta's experimental gene therapy and experiencing acute liver failure.

The firm will combine every 15 shares into one share in order to comply with Nasdaq's minimum bid price requirement.

While NICE recommended the drug for patients with at least one F508del mutation, Vertex struck a reimbursement deal with NHS England for a broader population.