Rare Disease Spotlight: Prader-Willi pipeline remains centered on obesity management

While the Prader-Willi syndrome pipeline spans a range of targets, most of the therapies in development address metabolic aspects of the disease, with few targeting its neurological manifestations, and none its root cause.

Prader-Willi syndrome (PWS) results from failure to express paternal genes in a region of chromosome 15, disrupting neurological and metabolic pathways. In healthy individuals, the maternal copy of the 15q11-q13 region is epigenetically silenced, while the paternal one is expressed. ...