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% ...

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 ...

Traditional academic training of early career researchers is often limited by the resources of the research group and the managerial strategy of the group PIs. Complexity72h is a workshop format ...

As well as directly causing or predisposing to disease, structural variants might function as susceptibility alleles in complex genetic ... of studies of the human dystrophin gene, one study ...