Foundation for Prader-Willi Blog (62)

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. Before drug candidates can be tested in humans, they need to show they are safe and efficac...

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

Dream, Believe, Achieve

I’m a mom, so you know my memory is full of as many holes as Swiss cheese. I can’t remember much of anything that took place more than 5 years ago. On particularly fine days, I may be lucky to remember something that was said within the past 5 minute...

January 13, 2017 Read More

Topics: Stories of Hope

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

A Dream I Can Believe In

When I was pregnant with Lily, I had all of these dreams of what it would be like to have a baby. I read all of the books on parenting I could get my hands on: books about what to expect while I was expecting, how to have the happiest baby on the blo...

January 7, 2017 Read More

Topics: Stories of Hope

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

FPWR Blog

Categories

De-Risking PWS Drug Development Through Preclinical Screening

Identifying the Neurobiology Behind Sleep Issues in PWS

PWS Research: Defining SNORD116 Gene Targets, Fixing Function

Dream, Believe, Achieve

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

The Value of Patient Input and Perspective in Drug Development

A Dream I Can Believe In

2016: Another Remarkable Year for Prader-Willi Research

PWS Clinical Trials Update - Part 2

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