With top-line pivotal data with gene therapy RGX-202 for Duchenne muscular dystrophy (DMD) due in the next quarter, Regenxbio Inc. rolled out positive interim data from the phase I/II Affinity trial ...

A study involving a small cohort of women who have received womb transplants has cast fresh light on how the immune system shapes pregnancy outcomes, opening up new avenues of research into ...

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 ...

Similarities among three pediatric brain tumors that arise in different structures of the CNS – pineoblastoma, retinoblastoma and Group 3 medulloblastoma – have been linked to their shared origin ...

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 ...

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Terns Pharmaceuticals Inc. has synthesized glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of diabetes, obesity, liver diseases and cardiometabolic syndrome.

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 ...

If one could sweep the brain clean and send the toxic substances that drive neurodegeneration to the recycling bin, perhaps one could treat Alzheimer’s disease (AD). Researchers at the Chinese Academy ...

The regulatory clouds that have been darkening the U.S. FDA landscape of late for Uniqure NV’s gene therapy AMT-130 in Huntington’s disease may be parting a bit with the announced departure of Vinay ...