NEW YORK – Royalty Pharma has acquired the remaining royalty interest of PTC Therapeutics in Roche's spinal muscular atrophy drug Evrysdi (risdiplam) for up to $300 million, the company announced ...

The NDA is supported by data from a single-arm, multi-center, open-label, Phase II registration study in China that completed enrollment of 87 patients in March.

Patients submitted for consideration for treatment with GS010/Lumevoq must meet specific eligibility criteria, including the length of time since the onset of their vision loss.

This year, there was a surge of new lung cancer treatments, more options for patients with earlier-stage disease, and growing ...

While autologous therapies are mainstream in blood cancers, experts warn against overhyping allogeneic and in vivo approaches ...

The Recommended Uniform Screening Panel now includes DMD and MLD, which has implications for firms developing treatments for ...

Jacobio will get $100 million upfront in the deal and be entitled to an additional $1.92 billion upon meeting certain milestones related to JAB-23E73.

NEW YORK – The convergence of gene-editing breakthroughs and new regulatory frameworks signaled a turning point in 2025 for getting gene therapies for rare diseases to patients. It's notoriously ...

NEW YORK – Daiichi Sankyo and AstraZeneca have begun dosing patients with HER2-expressing endometrial cancer in a Phase III trial testing whether the antibody-drug conjugate Enhertu (trastuzumab ...

NEW YORK – BioMarin has stopped developing the investigational BMN 349 for treating alpha-1 antitrypsin deficiency (AATD)-associated liver disease, the company announced in a regulatory filing this ...