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% ...

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 ...

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 ...

Dystrophin is a critical component of a protein complex that strengthens muscle fibers and protects them from injury during muscle contraction. Due to a genetic mutation in the DMD gene, people with ...

DMD is a complex disease, Kelly said ... for which it plans to pursue accelerated approval—based on FDA feedback—with dystrophin expression as a surrogate endpoint. In September 2024, the company ...

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 ...

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 ...

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 ...

Fans first heard the track on January 4, when Scott performed it during the College Football Playoff National Championship Halftime Show at Atlanta’s Mercedes-Benz Stadium. “Four-by-four, I ...

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