In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported ...

Solid Biosciences has surged back into the Duchenne muscular dystrophy (DMD) gene therapy race. | Solid Biosciences has ...

研究人员首次发现，人类细胞自带一套精密的"应急修复系统"，当检测到关键基因故障时，会自动激活"替补基因"展开修复。更令人振奋的是，这项机制可被人工干预精确调控，为DMD乃至6000多种单基因遗传病带来 ...

Day 90 biopsy data reported from first 3 participants dosed in Phase 1/2 INSPIRE DUCHENNE trial —— Average microdystrophin ...

All membranes were visualized using ECL Prime western blotting detection reagent (Cytive) and ChemiDoc (Bio-Rad). Band intensity and total protein normalization were determined using Image Lab ...

A screen for proteins that interact with β2-syntrophin led to the isolation of MAST205 (microtubule-associated serine/threonine kinase-205 kD) and a newly identified homologue, SAST (syntrophin ...

Background: Dystrophin is a key protein encoded by the DMD gene, serves as a scaffold linking the cytoskeleton to the extracellular matrix that plays a critical role in muscle contraction, relaxation, ...

Solid Biosciences (SLDB) announced initial data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy ...