SAN FRANCISCO, Nov. 21, 2017 /PRNewswire/ -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living ...

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 ...

Myotubular myopathy is an X-linked genetic disease affecting 1 in 50,000 newborn boys. It is caused by mutations in the MTM1 gene encoding myotubularin, a protein involved in the functioning of muscle ...

A collaborative research team successfully mitigated some of the effects of a muscular disease by using a new targeted enzyme replacement therapy strategy from 4s3 Bioscience. The team was inclusive ...

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 ...

DBMR has added a new report titled Myotubular Myopathy Treatment Market with data Tables for historical and forecast years represented with Chats & Graphs spread through Pages with easy to understand ...

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 ...

RYR1 mutations have been mainly implicated in CNM2 associated with autosomal-recessive inheritance. 3, 4 Implication of heterozygous RYR1 mutations in dominant CNM awaits confirmation. MTMR14/hJUMPY ...

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 ...

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, ...