Foundation for Prader-Willi Blog

Study Ties PWS Characteristics to PC1 Enzyme Deficiency [VIDEO]

FPWR is excited to share a new discovery in Prader-Willi Syndrome! Columbia University Medical Center researchers Lisa Burnett, PhD, and Rudolph Leibel, PhD, have published a breakthrough discovery in the Journal of Clinical Investigation that change...

February 2, 2017 Read More

Topics: Research

De-Risking PWS Drug Development Through Preclinical Screening

Any drug development process proceeds through several stages in order to produce a drug that is safe, efficacious, and has passed all regulatory requirements. The discovery phase converts what we have learned about the causes and the biology of the d...

January 25, 2017 Read More

Topics: Research

Identifying the Neurobiology Behind Sleep Issues in PWS

Sleep disturbances and daytime sleepiness severely impact the quality of life for many with PWS, as well as their parents and caregivers. Within the Global PWS Registry, about 50% of respondents report that their loved one suffers from excessive dayt...

January 23, 2017 Read More

Topics: Research

PWS Research: Defining SNORD116 Gene Targets, Fixing Function

January 16, 2017 Read More

Topics: Research

Studying a Novel Model of Adult-Onset SNORD116 Deletion in Mice

In contrast to many rare diseases that can be traced to a single gene, PWS involves a region of DNA that encompasses numerous genes. A lot of PWS research is centered around trying to connect the dots between the genes in the PWS region and the actua...

January 11, 2017 Read More

Topics: Research

The Value of Patient Input and Perspective in Drug Development

Janet Woodock, MD, Director of the Center for Drug Evaluation and Research at the FDA, recently highlighted how the FDA is placing a high priority on patient input and perspective in the drug development process. The agency recognizes that patients, ...

January 9, 2017 Read More

Topics: Research

2016: Another Remarkable Year for Prader-Willi Research

FPWR community, 2016 was yet another remarkable year for Prader-Willi research! Our supporters have allowed FPWR to fund more research than ever before, taking us closer to treatments for our loved ones with Prader-Willi syndrome. Read below for a fe...

January 6, 2017 Read More

Topics: Research

PWS Clinical Trials Update - Part 2

Over the past 3 years we have seen a number of new companies investing in Prader-Willi Syndrome. This is in part because of PWS's orphan indication and the significant unmet medical need, but is also reflective of the steps we have taken as a communi...

January 4, 2017 Read More

Topics: Research

PWS Clinical Trials Update - Part 1

Over the past 3 years we have seen a number of new companies investing in Prader-Willi Syndrome. This is in part becuase of PWS's orphan indication and the significant unmet medical need, but is also reflective of the steps we have taken as a communi...

January 2, 2017 Read More

Topics: Research

Foundation for Prader-Willi Blog | Research (32)

Categories

Study Ties PWS Characteristics to PC1 Enzyme Deficiency [VIDEO]

De-Risking PWS Drug Development Through Preclinical Screening

Identifying the Neurobiology Behind Sleep Issues in PWS

PWS Research: Defining SNORD116 Gene Targets, Fixing Function

Studying a Novel Model of Adult-Onset SNORD116 Deletion in Mice

The Value of Patient Input and Perspective in Drug Development

2016: Another Remarkable Year for Prader-Willi Research

PWS Clinical Trials Update - Part 2

PWS Clinical Trials Update - Part 1

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