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.

Endometrial02, researchers want to see whether Enhertu can replace current standard-of-care chemotherapy for patients with HER2-expressing tumors.

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 ...

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.

RheumaGen will use the funds to advance its therapeutic development program in celiac disease through preclinical proof of concept.

Now, Musunuru and colleagues are preparing a master clinical protocol to study a CRISPR therapy that's mostly the same as the one they used to treat baby KJ, except they plan to swap out the gene ...