FPWR Blog

Dad Believes In an Amazing Life For His Son With PWS

A special contribution by Brian Kalasek

My introduction to PWS probably started very similar to other PWS families. We were admitted to the NICU on day 3 of Paxton’s life. He hadn’t really “woken up” yet, but the nurses and doctors up to that point didn’t seem too concerned. They kept telling us he had WWBS, Wimpy White Boy Syndrome. Funny I thought, I’ve been fighting that for the past 36 years myself with limited success. By the end of the day 3 though everything had changed, we went from what we thought was a healthy little boy in the morning to total despair by dinner time.

Topics: Stories of Hope

GDF15: A New Appetite-Regulating Pathway With PWS Therapy Potential

A series of recent discoveries have defined a new pathway for regulating appetite and satiety suggesting that a naturally occurring protein in our bodies could have the potential to be a safe and effective therapy for obesity. This protein, known as growth differentiation factor 15 or GDF15 (or MIC1), is mostly made in the liver but also in other tissues. It normally circulates in the blood and functions in many cellular processes such as inflammation, response to stress, energy expenditure etc. However, its levels can increase in several disease states including cardiovascular disease, liver disease, diabetes, dementia and especially advanced cancer.

Topics: Research

Two Moms, 5 Years and $1.7M for PWS Research

Becky McWilliams and Samantha (Sam) Chipetz are a force to be reckoned with! In addition to managing successful careers and juggling a family, therapy appointments and play dates, these two hard working moms have been fundraising for PWS research for more than 5 years! Dedicated to finding treatments for the challenges that their sons, Brandon and Kieran, face on a daily basis, Sam and Becky have hosted walks, galas and other fundraising activities which have collectively raised over $1.7 million! 

Topics: News

PWS Clinical Trial: Effects Of GLWL-01 On Hyperphagia, Phase 2

GLWL Research is now recruiting patients for its Phase 2 study to evaluate the efficacy, safety and pharmacokinetics of GLWL-01 in the treatment of patients with Prader-Willi syndrome. The study will compare the effects of GLWL-01 on hyperphagia as compared to a placebo. You can learn more about GLWL-01 in the video below, from the clinical trials session at the 2017 FPWR conferece:

Topics: Research

Dr. Nectow Researches Role of DRN Brain Region and Hyperphagia in PWS

Researchers throughout the world are hard at work trying to identify the biological mechanism(s) that drive hyperphagia (excessive eating) in PWS. One of these scientists is Dr. Alexander Nectow at Princeton Univeristy.

Dr. Nectow's group studies a region of the brain called the dorsal raphe nucleus (DRN). Two populations of neurons in the DRN help control food intake and can cause animals to either binge eat or to stop eating.

 

Topics: Research

Intellectual Disability and PWS Researcher Godler Awarded $500,000

FPWR–funded researcher Dr. David Godler, of Murdoch Children’s Research Institute and University of Melbourne in Australia, was recently awarded nearly $500,000 AUD from the Turnbull Government’s Medical Research Future Fund (MRFF). The award funds a four-year project to further his work investigating the significance of mosaicism to intellectual disability in pediatric disorders. Dr. Godler's work and that of other grantees is described in this news release.

Topics: Research

You Do You! New 'Take Action' PWS Fundraising Tool

When it comes to fundraising for PWS research, I've got good news, and I've got good news: First, in 2017, our community raised $3,200,000 for PWS research! Thanks to everyone who fundraised or supported a fundraiser, last year FPWR funded 18 research grants, 2 novel drugs, 2 medical devices, 4 clinical trials, 6 models of PWS! 

Now for the good news: to kick off 2018, we've launched a new Take Action page on our website to give you more ways to support PWS research, including:

Topics: News

PWS Registry Data: Developmental Milestones in PWS [INFOGRAPHIC]

Since the launch of the Global PWS Registry in 2015, nearly 1,400 participants have enrolled. This month, the registry is highlighting results from the developmental milestones survey. This survey is aimed at understanding the age individuals with PWS reach various milestones. The survey includes physical milestones such as sitting, crawling, and walking, as well as social and cognitive milestones such as first words and reading.

Topics: Research

Welcome FPWR's New CEO, John Walter

Please join me in welcoming FPWR's newest team member, Chief Executive Officer John Walter!

Walter has dedicated his entire professional career to the non-profit sector, with over 30 years of experience in the field. Former CEO of the Leukemia and Lymphoma Society, Walter is an accomplished leader with a proven track record of building successful organizations. At FPWR, he'll play a crucial role in our next phase of growth and will work with the FPWR team to accelerate research and the development of therapies for PWS.

Topics: News

Dr. Dimitropoulos Uses Telehealth To Bring PWS Behavioral Therapies Home

There are numerous efforts in the healthcare community to increase health care accessibility, education, and support. One of these approaches is “telehealth”, the use of telecommunications methods such as computers and phones to deliver health related services and information. Dr. Anastasia Dimitropoulos at Case Western University is working on a project to determine the feasibility and effectiveness of using telehealth to bring behavioral therapies and interventions to families of those with PWS. They are working on two interventions.

Topics: Research

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