The US Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to NS-051/NCNP-04, which is being developed for the treatment of Duchenne muscular dystrophy (DMD) by Japanese drugmaker Nippon Shinyaku (TYO: 4516).
The FDA’s Rare Pediatric Disease designation is granted for treatments intended for serious or life-threatening diseases that affect children under the age of 18 and less than 200,000 patients in the USA.
DMD is a progressive muscle dystrophy caused by deficiency of the dystrophin protein leading to weakness of skeletal, cardiac and pulmonary muscles. There are many types of genetic mutations in DMD, and NS-051/NCNP-04 targets to treat DMD patients with confirmed gene mutations amenable to exon 51 skipping therapy.
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