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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.

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New resources to support Teva Pharmaceutical Industries' Tjet injector system...

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...

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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...

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PerkinElmer announces new Signature Precision Panel | Prenatal diagnostic...

PerkinElmer, Inc., a global leader focused on the health and safety of people and the environment, today announced that the Company's Signature Genomics Laboratories has launched its new Signature...

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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...

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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...

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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...

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

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...

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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.

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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...

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

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,...

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Scientists report major breakthrough in understanding molecular basis for...

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...

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‘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.

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

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...

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Alizé Pharma completes two Phase I clinical trials of AZP-531 in healthy...

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...

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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.

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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...

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Alizé Pharma announces launch of AZP-531 Phase II trial in patients with...

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...

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Rhythm, Actavis announce initiation of relamorelin Phase 2b trial for...

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...

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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...

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