There is broad agreement that psychiatric diagnoses in their current form are not reflective of any underlying biology, and that this is one of the things hampering psychiatric drug development. “We ...
Drugs that mimic GLP-1 are widely used to treat diabetes and obesity, but it is not fully understood exactly how they produce many of their beneficial effects. Investigators from The Salk Institute ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
Pepgen Inc. is forging ahead with tests of PGN-EDODM1 in other territories after the U.S. FDA placed a partial hold on the ...
What Cowen analyst Tara Bancroft called an “exciting” year ahead for Day One Biopharmaceuticals Inc. will be shared by Servier SAS, after the French firm agreed to pay $21.50 per share to acquire Day ...