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FDA gives third rare-disease drug another shot, Regenxbio says
The Food and Drug Administration has agreed to reverse its rejection of Regenxbio’s experimental gene therapy for a rare and fatal brain disease, the company said, the latest about-face by the agency ...
FDA drug approval affects healthcare around the world, but political shortcuts could hurt the agency’s international reputation
At the beginning of July 2026, the U.S. Food and Drug Administration approved the first gene therapy for young children with ...
FDA approves at-home starter dose of Eisai-Biogen Alzheimer's drug
The U.S. FDA on Monday approved an at-home starting dose of Eisai's and Biogen's Alzheimer's drug, allowing some ​patients to ...
US FDA approves Celcuity's breast cancer drug
July 14 (Reuters) - The U.S. FDA has approved Celcuity's drug for an advanced form of breast cancer, the regulator said on Tuesday, making it the company's first product to gain market entry and ...
FDA approves Nashville doctor's potential cure for sickle cell disease
A Nashville doctor helped pioneer an FDA approved "state-of-the-art" gene-editing therapy that could be a cure for painful ...
Science Daily
FDA-approved drug may finally help immunotherapy defeat rare liver cancer
Researchers found that a rare liver cancer evades immunotherapy by luring immune T cells away from the tumor and trapping them in nearby fibrous tissue. An FDA-approved drug called AMD3100 freed those ...
Newsweek
FDA U-Turns on Rare Disease Treatment: What it Means for Patients
See more of our trusted coverage when you search. Prefer Newsweek on Google to see more of our trusted coverage when you search. The U.S. Food and Drug Administration (FDA) has changed its stance on a ...
Wall Street Journal
FDA Gives Third Rare-Disease Drug Another Shot, Regenxbio Says
The Food and Drug Administration has agreed to reverse its rejection of Regenxbio’s RGNX1.28%increase; up pointing triangle experimental gene therapy for a rare and fatal brain disease, the company ...
CNBC
UniQure to seek FDA approval for Huntington's disease gene therapy after previous clash with agency
UniQure said it plans to seek approval for its experimental gene therapy for Huntington's disease. The announcement comes months after UniQure became embroiled in a public debate with FDA leaders over ...
USA Today
US FDA Has Granted A “Rare Pediatric Disease Drug” Designation for NV-387 As A Treatment for Measles – Enables Priority Review Voucher
SHELTON, CT / ACCESS Newswire / June 29, 2026 / NanoViricides, Inc., a publicly traded company (NYSE American:NNVC) (the “Company”), and a clinical stage, leading global pioneer in the development of ...