After Sarepta reported the death of a patient who had recently taken the gene therapy Elevydis, patient advocacy group Parent ...
GlobalData on MSN16h
Regenxbio’s DMD gene therapy drives 122.3% biomarker uptick in three-year-oldThe interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...
The death of a 16-year-old boy evokes a lengthy battle to realize the benefit of cutting-edge gene therapy while also ...
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
Sportschosun (English) on MSN18h
New Treatment Strategy for Rare Disease Dutsenic Muscle Dystrophy...EZH2 inhibitor steroid effective in combinationKorean researchers have proposed a new treatment strategy that can increase the effectiveness of the genetic rare disease ...
ROCKVILLE, MD, USA I March 19, 2025 I REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim data from two additional patients in the Phase ...
US gene therapy company Regenxbio has reported new, positive interim data from two additional patients in the Phase I/II ...
The money is developing treatments for diabetes, cancer and Alzheimer's disease. It could also mean a cure for a Ridgefield ...
U.S. Sen. Richard Blumenthal and researchers raise the alarm on how a federal medical research funding cap could impact "life ...
Sarepta stock may recover sharply as additional information and data reveal that the adverse event reported by the company is ...
Announced the U.S. FDA has accepted our Biologics License Application (BLA) seeking full approval of deramiocel for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathyBLA granted priorit ...
The warning comes amid reports that unregulated therapies are being offered directly to patients, potentially illegally.View ...
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