FDA Gives Approval For Duchenne Muscular Dystrophy Pediatric Treatment | Science 2.0
Nippon Shinyaku received rare pediatric disease designation for NS-050/NCNP-03, developed for Duchenne muscular dystrophy. NS-050/NCNP-03, an antisense oligonucleotide, aims to treat patients with gene mutations amenable to exon 50 skipping therapy, producing a functional dystrophin protein to suppress muscle function deterioration. Clinical trials are being prepared in Japan and the U.S.
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Neu-REFIX Beta glucan receives US FDA's Rare Pediatric Disease and Orphan Drug designations for treating Duchenne Muscular Dystrophy, aiding its progress from pre-clinical studies in Japan and India to clinical trials in the USA.
Nippon Shinyaku received rare pediatric disease designation for NS-050/NCNP-03, developed for Duchenne muscular dystrophy. NS-050/NCNP-03, an antisense oligonucleotide, aims to treat patients with gene mutations amenable to exon 50 skipping therapy, producing a functional dystrophin protein to suppress muscle function deterioration. Clinical trials are being prepared in Japan and the U.S.