References in periodicals archive ?
ASX: BLT; NASDAQ: BNTC; NASDAQ: BNTCW) has received US Food and Drug Administration orphan drug designation for BB-301, its DNA directed RNA interference (ddRNAi) therapeutic for the treatment of oculopharyngeal muscular dystrophy (OPMD), the company said.
Nuclear inclusions in oculopharyngeal muscular dystrophy consist of poly(A) binding protein 2 aggregates which sequester poly(A) RNA.
Nuclear accumulation of expanded PABP2 gene product in oculopharyngeal muscular dystrophy.