The strife-marked Duchenne muscular dystrophy (DMD) space drew forth another outspoken political figure in the shape of Sen. Ron Johnson (R-Wisc.), who said he was “enraged” by the U.S. FDA’s refusal ...

Scientists at Duke University have uncovered how macrophages help maintain intraocular pressure and have found that a specific type, resident macrophages, is essential for proper drainage of ...

Vertex Pharmaceuticals Inc. is on track to complete a BLA by the end of March seeking potential U.S. accelerated approval for povetacicept, its dual inhibitor of the BAFF and APRIL cytokines, to treat ...

Japan has approved the world’s first therapies derived from induced pluripotent stem cells (iPSCs), marking a major milestone for regenerative medicine and, potentially, a turning point in treating ...

If one could sweep the brain clean and send the toxic substances that drive neurodegeneration to the recycling bin, perhaps ...

Any lingering disappointment in the delay for Xenon Pharmaceuticals Inc.’s readout of the phase III X-Tole2 study testing azetukalner in focal onset seizures (FOS) appeared thoroughly extinguished as ...

In an article published in Cell Host & Microbe on March 3, 2026, researchers at McMaster University in Canada and at the Hospital Universitario de La Princesa in Spain have shed some light on the ...

Pepgen Inc. is forging ahead with tests of PGN-EDODM1 in other territories after the U.S. FDA placed a partial hold on the ...

In what it says could be the largest disclosed patent settlement in the pharmaceutical industry, Roivant Sciences Ltd. has reached a potential $2.25 billion settlement with Moderna Inc. over the use ...

In a boon for licensee Bristol Myers Squibb Co., Systimmune Inc.’s next-generation cancer drug, izalontamab brengitecan ...

CAR T cells have been groundbreaking for the treatment of B-cell cancers. But 8 years after Kymriah (tisagenlecleucel, ...

Targeting an ultrarare disease affecting about 250 U.S. residents, Immedica Pharma AB’s Loargys (pegzilarginase) won accelerated FDA approval to treat hyperargininemia in those ages 2 and older with ...