We hypothesized that the syntrophins function as molecular anchors to link dystrophin and utrophin to other cell structures. To understand the organization of the utrophin complex at neuromuscular ...

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 ...

Day 90 biopsy data reported from first 3 participants dosed in Phase 1/2 INSPIRE DUCHENNE trial —— Average microdystrophin expression of 110% ...

You may have heard that eating complex carbs is better than simple carbs. But nutrition labels don’t always tell you if the carbohydrate content is simple or complex. Complex carbohydrates are ...

The life expectancy of affected patients is significantly reduced. The disease, for which there is currently no cure, is caused by mutations in the dystrophin gene, which is located on the X ...

DMD is a complex disease, Kelly said, requiring a full toolbox of treatment options ... for which it plans to pursue accelerated approval—based on FDA feedback—with dystrophin expression as a ...

BioMarin Pharmaceutical Inc. ( NASDAQ: BMRN) Q4 2024 Earnings Conference Call February 19, 2025 4:30 PM ET Traci McCarty - Head of IR Alexander Hardy - President and CEO Brian Mueller - EVP and CFO ...

Mammoth Biosciences researchers have developed NanoCas, an ultracompact CRISPR nuclease, demonstrating its ability to perform ...

2020). We reasoned that the differential expression analysis of VL and TA in healthy individuals would provide insight into protective mechanisms relevant in the absence of dystrophin. The difference ...

Far from being solo operators, most single-celled microbes are in complex relationships. In the ocean, the soil, and your gut, they might battle and eat each other, exchange DNA, compete for ...