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.
Despite holding promise in a range of disease states, a variety of challenges prevent cell and gene therapies (CGTs) from more widespread use, according to a new report from Cardinal Health.
Delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) appeared to protect muscle from progressive damage in patients with Duchenne muscular dystrophy (DMD) based on muscle quantitative magnetic resonance measures.
Prademagene zamikeracel (Zevaskyn) is the first and only cell-based gene therapy for wound treatment in patients with recessive dystrophic epidermolysis bullosa.