Oliver and Amelia Carroll have Batten Disease - an incurable genetic disorder that causes rapid decline in a child's ability to walk, talk and see. | ITV News Granada ...
Tech firm Clario is boosting its electronic clinical outcome assessment (eCOA) offerings by acquiring the ...
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A 39-year-old father is suffering from a horrifically painful skin disease that doctors can't cure or even definitively ...
In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...
The FDA granted fast track designation to ATSN-201 for the treatment of X-linked retinoschisis, according to a press release from Atsena Therapeutics.
A mosquito-borne viral disease exceedingly rare in people killed a horse in Lackawanna County, though no human cases have ...
HEARTBREAKING details of the final act of loyalty shown to Gene Hackman by his dogs even after his death have been revealed.
GeneDx Holdings Corp. is delivering strong revenue growth, but it is still burning cash. Click here for more on WGS stock ...
The United States was the major funder of tuberculosis programs. Now hundreds of thousands of sick patients can’t find tests ...
Title: Initial Experience from the INSPIRE DUCHENNE Phase I/II Study of SGT-003 Microdystrophin Gene Therapy for Duchenne ...
On Feb. 27, 2025, Zevra entered into an asset purchase agreement for the sale of a Rare Pediatric Disease Priority Review Voucher (PRV), issued by the U.S. FDA upon the approval of MIPLYFFA, for $150 ...
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