New Natural History Study of Prader-Willi Syndrome Announced

According to a story from pm360online.com, a new study of Prader-Willi syndrome will soon be under way thanks to a recent collaboration between the biopharmaceutical company Zafgen and the Foundation for Prader-Willi Research. Zafgen hopes to use data from the study to inform the development of its proprietary drug candidate ZGN-1258, which is aimed at treating the syndrome. The Foundation for Prader-Willi Research (FPWR) is a non-profit organization dedicated to the curing and treatment of Prader-Willi syndrome through the support of drug development and disease research.

About Prader-Willi Syndrome

Prader-Willi syndrome is a genetic disorder that is characterized by the development of extreme childhood obesity, often accompanied by type 2 diabetes. In most cases, Prader-Willi syndrome is caused by the deletion of a certain portion of the father’s chromosome 15, and in others, the patient gets two copies of chromosome 15 from the mother, but none from the father. Symptoms of the syndrome vary throughout the life of the patient; at birth, symptoms include low muscle tone, poor feeding, and developmental delays. In childhood, patients are constantly hungry, sleep heavily, have some intellectual disability, have poor coordination, short stature, obesity, and delayed puberty. It is not inherited, as the genetic changes happen during fetal development. Treatment is primarily symptomatic, and most patients must have their access to food deliberately limited. To learn more about Prader-Willi syndrome, click here.

About The Study

The goal of the study is primarily to more thoroughly advance the knowledge of the natural history of the syndrome. This will involved observation of the medical history of patients and in-depth analysis of serious adverse events that appear as a result of Prader-Willi syndrome. The study will last for four years with enrollment slated to start in September 2018. Enrollment will be conducted with the Global Prader-Willi Syndrome Patient Registry, a valuable resource that is supported by the National Organization for Rare Disorders (NORD). 

Why Study Prader-Willi Syndrome?

Although there is a reasonable body of knowledge about Prader-Willi syndrome, the reality is that there are no treatments available that address the root cause of the syndrome. The study will be an opportunity to make new observations about the disease that could have major impacts on not only drug development, but future research approaches as well, such as the design of clinical trials.

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