Prader Willi Syndrome News and Research

RSS

Scientists discover unexpected functions of snoRNAs that explains cause of some diseases

Scientists have discovered unexpected functions of small nucleolar RNAs (snoRNAs) that explain the cause of some diseases.

7 Jun 2016

New UCL study highlights genes associated with schizophrenia and obesity

Genes involved in schizophrenia and obesity have been highlighted in a new UCL study, which could lead to a better understanding of the DNA variants which affect risk of these conditions and aid the development of improved strategies for prevention and treatment.

18 Oct 2015

FDA awards research grants to boost product development for patients with rare diseases

The U.S. Food and Drug Administration today announced it has awarded 18 new research grants totaling more than $19 million to boost the development of products for patients with rare diseases, which affect the lives of nearly 30 million Americans.

11 Oct 2015

Bariatric surgery results in substantial weight loss in overweight children with Prader-Willi syndrome

Obesity is a leading cause of complications and death in children suffering from Prader-Willi syndrome (PWS), yet there are few effective treatment options for these patients. In a new study published in Surgery for Obesity and Related Disease researchers found that bariatric surgery, specifically laparoscopic sleeve gastrectomy (LSG), resulted in substantial weight loss with no apparent adverse effect on growth in a small group of severely overweight patients with PWS.

29 Sep 2015

Rhythm Metabolic awarded FDA grant to advance ongoing setmelanotide clinical trial in Prader-Willi syndrome

Rhythm announced today the award of a $1 million research grant from the FDA Office of Orphan Products Development to its subsidiary, Rhythm Metabolic, Inc., to support the ongoing Phase 2 clinical trial of setmelanotide, the company's novel peptide MC4 agonist, for the treatment of obesity and hyperphagia in Prader-Willi syndrome (PWS).

24 Sep 2015

Rhythm Metabolic completes $40 million Series A financing

Rhythm announced today the completion of a $40 million Series A financing for its subsidiary, Rhythm Metabolic, Inc., which is developing setmelanotide (RM-493), a novel peptide MC4 agonist for the treatment of obesity caused by genetic deficiencies in the MC4 pathway.

13 Aug 2015

Rhythm announces initiation of two setmelanotide Phase 2 trials for treatment of PWS, POMC-null obesity

Rhythm announced today the initiation of two Phase 2 clinical trials focused on evaluating the safety and effectiveness of setmelanotide (RM-493), the company's novel melanocortin 4 receptor (MC4R) agonist, for the treatment of Prader-Willi Syndrome (PWS) and POMC-null obesity.

4 Jun 2015

Alizé Pharma presents clinical results from the AZP-531 program at the ADA 75th Scientific Sessions

Detailed data will be presented from the two Phase I trials conducted in 76 healthy volunteers and overweight or obese subjects...

26 May 2015

Johns Hopkins researchers link sperm with specific 'epigenetic tags' to autism

In a small study, Johns Hopkins researchers found that DNA from the sperm of men whose children had early signs of autism shows distinct patterns of regulatory tags that could contribute to the condition. A detailed report of their findings will be published online in the International Journal of Epidemiology on April 15.

15 Apr 2015

Rhythm, Actavis announce initiation of relamorelin Phase 2b trial for treatment of diabetic gastroparesis

Rhythm, a biopharmaceutical company, and Actavis plc, a leading global pharmaceutical company, announced today the initiation of a Phase 2b clinical trial assessing the efficacy and safety of relamorelin (RM-131), Rhythm's ghrelin agonist, for the treatment of gastroparesis in patients with type 1 and type 2 diabetes.

8 Apr 2015

Alizé Pharma announces launch of AZP-531 Phase II trial in patients with Prader-Willi syndrome

Alizé Pharma, a company specialized in the development of drugs for the treatment of metabolic disorders and rare diseases, today announces the launch of a Phase II clinical trial of AZP-531, its unacylated ghrelin analog, in patients with Prader-Willi syndrome.

3 Mar 2015

Study suggests link between neonatal ghrelin and obesity risk

Our subconscious motivation to eat is powerfully and dynamically regulated by hormone signals. The gut-derived hormone ghrelin is one such key regulator, promoting appetite through its effects on neurons in a small region of the brain called the hypothalamus.

21 Jan 2015

Growth hormone boosts muscle development in Prader-Willi syndrome

Randomised trial findings show a positive effect of growth hormone therapy on muscle thickness in infants with Prader-Willi syndrome.

22 Dec 2014

Alizé Pharma completes two Phase I clinical trials of AZP-531 in healthy volunteers, obese subjects

Alizé Pharma SAS, an Alizé Pharma group company specialized in the development of biopharmaceuticals to treat metabolic disorders and rare diseases, announces today the completion of two Phase I clinical trials with its unacylated ghrelin analog AZP-531 in 76 healthy volunteers and overweight or obese subjects.

10 Dec 2014

Dosing commences in Essentialis’ DCCR clinical study in obese Prader-Willi syndrome patients

Essentialis announced today the dosing of the first patient in clinical study PC025, which is designed to evaluate the safety and efficacy of diazoxide choline controlled release tablets (DCCR) in obese Prader-Willi syndrome patients.

27 Jun 2014

‘Surprising’ effect of growth hormone on BMI in normal-weight children

Researchers have found that the body mass index standard deviation score increases in normal-weight children receiving growth hormone treatment.

16 Jun 2014

Scientists report major breakthrough in understanding molecular basis for Prader-Willi syndrome

Scientists at the Hebrew University of Jerusalem have reported a major breakthrough in understanding the molecular basis for Prader-Willi syndrome (PWS), perhaps the most studied among the class of diseases that involves defects in parental imprinting.

14 May 2014

Alize Pharma initiates AZP-531 Phase I clinical trial for treatment of type 2 diabetes

Alize Pharma, a company specialized in the development of drugs for the treatment of metabolic diseases and rare diseases, announces today the launch of the first Phase I clinical trial for AZP-531, its unacylated ghrelin analog, in type 2 diabetes.

16 Jul 2013

Prader-Willi syndrome results in dysregulation of circadian and metabolic genes

Researchers with the UC Davis MIND Institute and Agilent Laboratories have found that Prader-Willi syndrome - a genetic disorder best known for causing an insatiable appetite that can lead to morbid obesity - is associated with the loss of non-coding RNAs, resulting in the dysregulation of circadian and metabolic genes, accelerated energy expenditure and metabolic differences during sleep.

26 Jun 2013

University of Alberta researchers help children with Down syndrome

Researchers from the University of Alberta are helping children with Down syndrome who stutter find their voice and speak with ease.

26 Feb 2013