Pushing for progress in Prader-Willi

How Prader-Willi patient advocates are getting in the weeds of drug discovery

In the wake of the 2015 clinical failure of Zafgen Inc.’s subcutaneous beloranib for obesity in Prader-Willi syndrome patients, the Foundation for Prader-Willi Research (FPWR) last year launched a five-year, $26 million strategic research plan that included an initiative to develop better preclinical models for the indication. With the program, FPWR joins a growing group of patient-centric disease organizations playing a pivotal and expanding role in supporting and de-risking potential therapeutics as they move through preclinical stages and into the clinic.

Prader-Willi is a rare genetic disease driven by the deletion or silencing of at least a dozen genes paternally expressed on chromosome 15, leading to anatomical changes in the hypothalamus that impact the musculoskeletal system, and cause hyperphagia leading to obesity. It affects about 200,000 people worldwide, and compounds in development have received orphan designation in the U.S. and the EU.

The disease involves

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