Sub Subramony, M.D., discusses the challenges faced by patients with Friedreich ataxia in accessing healthcare services and managing their condition, as well as the significant burden the disease places on families and caregivers.
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WVE-N531 Shows Promising Efficacy in DMD at Interim FORWARD-53 Analysis
September 26th 2024In the phase 2 FORWARD-53 study, the exon-skipping oligonucleotide WVE-N531 showed promising safety and efficacy in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping.
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