A team of researchers has demonstrated the efficacy of administration of a therapeutic vector by a single intravenous injection and identified the dose that restores long-term muscular strength in a ...

Ann & Robert H. Lurie Children's Hospital of Chicago is one of the few centers participating in ASPIRO, an international Phase 1/2 clinical trial of a gene therapy product called AT132 for X-linked ...

Work on gene therapy is showing significant progress for restoring muscle strength and prolonging lives in dogs with a previously incurable, inherited neuromuscular disease. UW Medicine Institute for ...

TOKYO, Nov. 15, 2023 /PRNewswire/ -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") today announced that The Lancet Neurology published a preliminary data analysis from ...

Tiny deviations in the body's cells can sometimes have severe consequences. Researchers have discovered why cells from patients suffering from the rare muscular disease myotubular myopathy cannot ...

SAN FRANCISCO--(BUSINESS WIRE)--Audentes Therapeutics, Inc., an Astellas company, today announced that a third study patient has passed away in the ASPIRO clinical trial evaluating AT132 in patients ...

The very recent report of deaths in a gene therapy trial for children with X-linked myotubular myopathy (MTM) is a tragic reminder of how difficult it is to predict outcomes in first-in-human studies.

The alternative text for this image may have been generated using AI. Contrary to what is indicated in OMIM, the reported mutation in MYF6 did not cause a recognized form of centronuclear myopathy. 2 ...

SAN FRANCISCO--(BUSINESS WIRE)--Audentes Therapeutics, an Astellas genetic medicines company, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold for the ...

Evry (France), April 5, 2017. A team of researchers in France, led by Dr. Ana Buj-Bello (Genethon/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, ...