Prader Willi Syndrome News and Research

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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

Prader-Willi children may receive relief from sleep disorders after adenotonsillectomy

Children with Prader-Willi syndrome may receive relief from sleep disorders after undergoing an adenotonsillectomy, suggests a new study from Nationwide Children's Hospital published in the November print issue of the Archives of Otolaryngology-Head and Neck Surgery.

21 Nov 2012

People with Prader-Willi Syndrome may have impaired autonomic nervous system

An Australian study reveals that people with the rare genetic disorder known as Prader-Willi Syndrome may have an impaired autonomic nervous system. This discovery opens up a new way of looking at the insatiable appetite experienced by all sufferers, as well as their very high risk of cardiovascular disease.

16 Nov 2012

Brains of people with anorexia and obesity are wired differently

The brains of people with anorexia and obesity are wired differently, according to new research. Neuroscientists for the first time have found that how our brains respond to food differs across a spectrum of eating behaviors - from extreme overeating to food deprivation.

4 Apr 2012

Oxytocin hormone holds promise for children with Prader-Willi syndrome

Prader-Willi syndrome is a rare genetic disorder which affects one child in 25,000. Children born with this syndrome have a range of complex neurological and developmental problems which continue into adult life. These can manifest as cognitive and behavioral difficulties, weight gain, problems in controlling their temper and attendant difficulties in socialization.

24 Jun 2011

Scientists discover simple molecular test to block GOAT enzyme

Scientists at The Scripps Research Institute have designed a new molecular test that will allow researchers to look for potential drugs targeting a human metabolic enzyme believed to stimulate the appetite and play a role in diabetes.

17 Sep 2010

New resources to support Teva Pharmaceutical Industries' Tjet injector system now available

Teva Pharmaceutical Industries Ltd. announced today the availability of new resources to support the recently introduced Tjet® injector system, a needle-free device designed to deliver Teva’s TEV-TROPIN® [somatropin (rDNA origin) for injection] brand human growth hormone (hGH) to children who have growth failure due to inadequate secretion of normal endogenous growth hormone.

3 Feb 2010

Research to explore diagnosis and treatment of rare diseases

The National Institutes of Health announced today a second phase of the Rare Diseases Clinical Research Network (RDCRN) including funds for 19 research consortia.

7 Oct 2009

NORD urges members of Congress to pass comprehensive health reform legislation

On behalf of the 30 million Americans who are affected by one of the nearly 7,000 rare disorders, the National Organization for Rare Disorders (NORD) urges you to pass comprehensive health reform legislation to assure that every American has access to affordable, meaningful health insurance coverage. Failure to pass such long-overdue legislation this year would be devastating for people with rare diseases, and for millions of others burdened by chronic illness.

15 Sep 2009

Analysis suggests human development is set by ongoing interplay of parent and offspring genes

An analysis of rare genetic disorders in which children lack some genes from one parent suggests that maternal and paternal genes engage in a subtle tug-of-war well into childhood, and possibly as late as the onset of puberty.

28 Jul 2009

New technique provides closer look at DNA and mRNA

In a study in the advance online edition of Nature Structural and Molecular Biology, researchers from Albert Einstein College of Medicine describe a technique for looking more precisely at a fundamental step of a cell's life - a gene, DNA, being read into a message, mRNA.

3 Dec 2008

Prader Willi Syndrome News and Research

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

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

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

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

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

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

University of Alberta researchers help children with Down syndrome

Prader-Willi children may receive relief from sleep disorders after adenotonsillectomy

People with Prader-Willi Syndrome may have impaired autonomic nervous system

Brains of people with anorexia and obesity are wired differently

Oxytocin hormone holds promise for children with Prader-Willi syndrome

Scientists discover simple molecular test to block GOAT enzyme

New resources to support Teva Pharmaceutical Industries' Tjet injector system now available

Research to explore diagnosis and treatment of rare diseases

NORD urges members of Congress to pass comprehensive health reform legislation

Analysis suggests human development is set by ongoing interplay of parent and offspring genes

New technique provides closer look at DNA and mRNA

Boys are twice as likely as girls in the U.S. and Asia to receive growth hormone

Prader-Willi syndrome - new genetic clues

FDA approves Norditropin for short stature in children with Noonan syndrome

Industry Focus eBook - Analytical Chemistry (2nd Edition) eBook

Bringing real-time analytics to downstream bioprocess monitoring

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

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