FDA removes clinical hold on Entrada’s Duchenne therapy
Entrada Therapeutics can plough on with the development of its Duchenne muscular dystrophy (DMD) therapy, ENTR-601-44, now ...
Nature22 天
Interactions between β2-syntrophin and a family of microtubule-associated serine/threonine ...
Understanding how the dystrophin and utrophin protein networks interact with other cellular components may reveal novel characteristics of cytoskeletal function and adaptability. We hypothesized ...
Solid says early data suggest ‘differentiated’ Duchenne gene therapy
The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including ...
来自MSN7 个月
New Gene Therapy Approach Improves Muscle Strength in DMD Mice
Duchenne muscular dystrophy is a genetic disorder caused by mutations in the DMD gene, resulting in a lack of the protein dystrophin ... recently approved a micro-dystrophin gene therapy for ...
pharmaphorum19 小时
Summit shares plunge nearly 80% after DMD drug fails
Ezutromid was designed to treat DMD by boosting utrophin levels to compensate for the faulty dystrophin protein that is the ... s results of AAVrh74.MHCK7.micro-Dystrophin trial.
The Scientist6 年
Infographic: Treating Duchenne Muscular Dystrophy with CRISPR
Dystrophin is part of a protein complex linking the cytoskeleton of muscle fibers to the surrounding connective tissue (basal lamina). It’s a long protein with numerous redundant coils (purple balls), ...