Gene Therapy

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While patients should be closely monitored in the first 90 days post-treatment, delandistrogene moxeparvovec showed a manageable, consistent safety profile in a broad population of patients. | Image Credit: Hypnosis - stock.adobe.com
Delandistrogene Moxeparvovec Shows Long-Term Safety, Tolerability in Pooled Trial Data

May 16th 2025

Delandistrogene moxeparvovec demonstrated a manageable safety profile across clinical trials for Duchenne muscular dystrophy (DMD), with most adverse events emerging within 90 days of infusion.

The new report highlights several barriers to CGT use, including cumbersome referral processes, high price tags, complex logistics, and uncertainties around reimbursement. | Image Credit: Treecha - stock.adobe.com
CGTs Have Potential to Transform Care, but Challenges Persist

May 14th 2025

Quantitative magnetic resonance imaging seemed to show that delandistrogene moxeparvovec protects muscle from progressive damage in DMD. | Image credit: OlegKachura - stock.adobe.com
Quantitative Magnetic Resonance Outcomes Suggest Gene Therapy Slows DMD Progression

May 14th 2025

gene therapy | Image Credit: © RFBSIP - stock.adobe.com
Expanding Broader Patient Access to CGTs

April 30th 2025

At 6 months, 81% of wounds showed 50% or more healing vs 16% of 43 matched control wounds treated with standard of care in the VIITAL study. | Image credit: wladimir1804 - stock.adobe.com
FDA Approves Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa

April 29th 2025

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