Genethon Reports Results of GNT0004 Gene Therapy Clinical Trial: Permanent Decrease in CK Levels of More Than 75%
Safety, efficacy, and pharmacodynamic results show good tolerance of GNT0004 associated with transient immunological prophylactic treatment, as well as efficacy data in terms of...
Precision BioSciences PBGENE-DMD Restores Production of Nearly Full-Length Dystrophin Protein
Precision BioSciences, today announced the strategic prioritization and acceleration of PBGENE-DMD, the Company’s first-in-class in vivo gene editing approach for Duchenne Muscular Dystrophy (DMD).
What...
Satellos Reports SAT-3247 Q1 2025 Results, Highlights Recent Company Progress and Future Plans
Satellos Bioscience, today announced its financial results and corporate highlights for the first quarter ended March 31, 2025.
“We are very pleased with our continued...
Sarepta Therapeutics Announces Approval in Japan of ELEVIDYS, a Gene Therapy to Treat Duchenne Muscular Dystrophy
People aged 3 to under 8 who do not have deletions in exons 8 and/or 9 of the DMD gene and who test negative...
SonoThera Ultrasound-Mediated Delivery Method Can Deliver Full-Length Dystrophin to Cells
The findings of the study, which are currently being developed for SonoThera's Duchenne Muscular Dystrophy (DMD) treatment, demonstrate the capability of Ultrasound-Mediated Delivery technology...