Vanda Pharmaceuticals Announces FDA Rare Pediatric Disease Designation for Investigational Therapy for Charcot-Marie-Tooth Disease Type 2S

Vanda Pharmaceuticals received Rare Pediatric Disease Designation from the FDA for VCA-894A, an investigational antisense oligonucleotide therapy designed to treat Charcot-Marie-Tooth disease type 2S. The designation applies to a serious and progressive neuromuscular disorder that primarily affects individuals from birth through age 18 and has an estimated prevalence of less than 1 in 1,000,000 worldwide.

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