A five-year-old boy who received the world's most expensive drug as a baby has made "incredible progress" and can walk ...

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 ...

Medcare Women & Children Hospital has successfully administered a pioneering intrathecal gene therapy for Spinal Muscular ...

Fakeeh University Hospital is emerging as a global destination for advanced gene therapy, delivering highly specialized ...

Royalty Pharma Plc (NASDAQ: RPRX) shares rose 1.5% in after-hours trading on Monday after the company announced it had ...

Investing.com -- Royalty Pharma Plc (NASDAQ:RPRX) stock gained 1.5% in after-hours trading Monday following the company’s announcement that it has acquired the final portion of PTC Therapeutics ’ ...

NEW YORK, Dec. 29, 2025 (GLOBE NEWSWIRE) -- Royalty Pharma plc (Nasdaq: RPRX) today announced that it has acquired the final portion of PTC Therapeutics’ remaining royalty on Roche’s Evrysdi for $240 ...

Abu Dhabi marks a landmark achievement in becoming the first in the world to deliver ITVISMA (onasemnogene abeparvovec) for ...

BHVN’s investment case definitely weakened after Opakalim’s Phase 2 MDD miss. This also means they’ll discontinue their ...

Inspired by his father, Grandview High School student Josue Clara started a nonprofit called Beyond Rare to build awareness ...

The Emirates Drug Establishment (EDE) has announced the approval of Itvisma (onasemnogene abeparvovec), a gene therapy that uses an adeno-associated viral vector to treat spinal muscular atrophy (SMA) ...

Novartis has discontinued two undisclosed programs under its current partership with Voyager, the biotech announced last month. Projects under the deal for spinal muscular atrophy and Huntington’s ...