When used for the appropriate seizure type, antiseizure medications (ASMs) are roughly equivalent in efficacy. In addition to the seizure type, the choice of drug is usually based on factors such as ease of use, spectrum of activity, adverse effects, interactions with other drugs, presence of comorbid conditions, suitability for elderly persons and those with childbearing potential, and cost. Treatment should begin with a single drug, increasing the dosage gradually until seizures are controlled or adverse effects become unacceptable. If seizures persist, expert clinicians generally try at least one and sometimes a second alternative drug as monotherapy...
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RELEASE
The adeno-associated virus (AAV) vector-based gene therapy delandistrogene moxeparvovec-rokl (Elevidys – Sarepta) received accelerated approval from the FDA in 2023 for treatment of ambulatory children 4-5 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.1 It has now received full approval for use in ambulatory patients ≥4 years old and accelerated approval for use in nonambulatory patients with DMD.
MECHANISM OF ACTION — Delandistrogene moxeparvovec is a nonreplicating, recombinant AAV vector-based therapy that delivers a transgene encoding microdystrophin protein, a shortened protein that contains selected domains of the dystrophin protein expressed in normal muscle cells.
CLINICAL STUDIES — FDA approval of the expanded indications were based on the results of earlier trials in …