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DMD Gene Therapy Shows Clinical Benefits in 8- and 9-Year-Old Patients

The findings support the clinical benefits of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD). Patients with Duchenne muscular dystrophy (DMD) aged 8 and 9 years experienced significant functional benefits with delandistrogene moxeparvovec-rokl (Elevidys; Sarepta Therapeutics) in part 2 of the EMBARK study (NCT05096221), according…

ajmc.com
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Duchenne Muscular Dystrophy

An FDA committee voted 9-3 that available data did not provide substantial evidence of effectiveness for deramiocel in DMD. Long-Term Data Reinforce Givinostat Efficacy, Safety in DMD DYNE-251 Granted FDA Breakthrough Therapy Designation for DMD FDA Recommends Sarepta Resume DMD Gene Therapy…

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