Gene Therapy

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Real-world data suggest delandistrogene moxeparvovec is well-tolerated and effective in patients with DMD. | Image credit: natali_mis - stock.adobe.com
Real-World Study Suggests DMD Gene Therapy Is Safe, Effective

March 26th 2025

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found tolerable and showed signs of efficacy in a real-world cohort.

Delandistrogene moxeparvovec is an adeno-associated virus vector-based gene therapy approved for the treatment of DMD. Image credit: natali_mis - stock.adobe.com
Delandistrogene Moxeparvovec Shows Long-Term Impacts at 3 and 5 Years in DMD

March 21st 2025

Just 4 of the 20 cell and gene therapy trials in the study submitted the same trial evidence to both the FDA and EMA. | Image credit: Treecha - stock.adobe.com
Uniform Global Standards, Processes Could Accelerate CGT Approvals, Access

March 13th 2025

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Genetic Hearing Loss Improves With DB-OTO Gene Therapy: CHORD Trial

February 27th 2025

In the INSPIRE DUCHENNE trial, interim 90-day biopsy data showed an average microdystrophin expression of 110% among the first 3 participants in the study. | Image credit: RFBSIP - stock.adobe.com
SGT-003 Gene Therapy Shows Potential in DMD

February 26th 2025

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