Solid Biosciences' SGT-003 gene therapy showed strong dystrophin expression, muscle health improvements, and no serious safety concerns in early trial data.
The scientists demonstrate that it is possible to specifically deliver the treatment to the muscle stem cells, avoiding ...
Physical therapy can help maintain muscle ... MedlinePlus. DMD gene. MedlinePlus. Duchenne muscular dystrophy. National Human Genome Research Institute. About Duchenne muscular dystrophy.
The trial was able to elicit more than double the microdystrophin gene in patients after 90 days of treatment.
Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | ...
Solid Biosciences’ gene therapy uses a shortened version of the dystrophin protein, which is engineered to mimic the key functions of full-length dystrophin and allows it be delivered more effectively ...
Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the ...
Pfizer’s decision to halt further marketing of Beqvez is further sign of the sparse patient interest in gene therapies for ...
Entrada Therapeutics is finally able to get its Duchenne muscular dystrophy candidate (DMD) back on track after the FDA ...
Entrada Therapeutics gains FDA clearance for its Phase 1b ELEVATE-44-102 study, evaluating ENTR-601-44 in adult Duchenne ...
Boston, USA-based biotech Solid Biosciences saw its shares rocket 36% to $5.48 yesterday, after the precision genetic ...
Average microdystrophin expression of 110% (N=3) and significant improvements in multiple additional muscle health biomarkers observed support the potential of SGT-003 as a next-generation, ...