(Press-News.org) A molecule created at the University of Auckland is one step closer to becoming a treatment for an extremely rare and severely debilitating neurological disorder called Phelan-McDermid syndrome. Children with the disorder showed significant improvements in a phase two clinical trial in the US, Neuren Pharmaceuticals, which is listed on the Australian Securities Exchange, said in December.
Next steps would be a phase three trial and seeking approval from the US Food & Drug Administration. The molecule, NNZ-2591, comes from work years ago by Dr Jian Guan and Professor Sir Peter Gluckman at the Liggins Institute and Distinguished Professor Dame Margaret Brimble in her chemistry laboratory.
That’s the same group whose work led to the breakthrough drug Trofinetide, approved last year for treating Rett syndrome, another rare genetic neurological disorder. The treatment is marketed under the name Daybue.
Neuroprotective qualities of a peptide called cyclic glycine-proline (cGP) were investigated by Guan and Gluckman. In Brimble’s lab, Dr Paul Harris produced NNZ-2591, a synthetic analogue of cGP, which Neuren Pharmaceuticals hopes will become a Phelan-McDermid treatment. The University spun out a company to commercialise research, ultimately selling out.
Intellectual disability, low muscle tone, developmental delays and symptoms of autism can be among the features of Phelan-McDermid.
Neuren is also conducting Phase 2 clinical trials of NNZ-2591 in children with three other neurodevelopmental disorders – Pitt Hopkins syndrome, Angelman syndrome and Prader-Willi syndrome. Likewise, NNZ-2566, the Trofinetide/Daybue molecule, may eventually be used for disorders beyond Rett syndrome.
END
New molecule from University step closer to treatment for rare disease
A molecule created at the University of Auckland is one step closer to becoming a treatment for an extremely rare and severely debilitating neurological disorder called Phelan-McDermid syndrome.
2024-02-02
ELSE PRESS RELEASES FROM THIS DATE:
Machine learning to battle COVID-19 bacterial co-infection
2024-02-02
University of Queensland researchers have used machine learning to help predict the risk of secondary bacterial infections in hospitalised COVID-19 patients.
The machine learning technique can help detect whether antibiotic use is critical for patients with these infections.
Associate Professor Kirsty Short from the School of Chemistry and Molecular Biosciences said secondary bacterial infections can be extremely dangerous for those hospitalised with COVID-19.
“Estimates of the incidents of secondary bacterial infections in COVID-19 ...
Some tumors ‘grow bad’: Why a dangerous subtype of Wilms tumor is so resistant to chemotherapy
2024-02-02
An international team, led by researchers at Nagoya University in Japan, may have determined why the diffuse anaplasia (DA) subtype of Wilms tumor (WT) resists chemotherapy. This subtype grows even when it has a high burden of DNA damage and increases the mutation rate of tumor protein 53 (TP53), a gene that plays a critical role in the regulation of cell growth and division. The team’s findings, published in Modern Pathology, suggest new ways to treat this subtype.
WT, also known as nephroblastoma, is the most common childhood cancer originating in the kidney. Fortunately, the survival rate of adolescents ...
Disrupted cellular function behind type 2 diabetes in obesity
2024-02-02
Disrupted function of “cleaning cells” in the body may help to explain why some people with obesity develop type 2 diabetes, while others do not. A study from the University of Gothenburg describes this newly discovered mechanism.
It is well known that obesity increases the risk of insulin resistance and type 2 diabetes. It is also well known that some people who gain weight suffer from the disease and others do not. The reasons for these differences are not clear, but they are related ...
USC launches School of Advanced Computing
2024-02-02
USC President Carol Folt launched the university’s first new school in more than a decade: the USC School of Advanced Computing, a cornerstone of her $1 billion advanced computing initiative. The school seeks to educate all students, regardless of their major, in the ethical use of computing technology as part of the president’s Frontiers of Computing initiative.
Gaurav Sukhatme — a professor of computer science and electrical and computer engineering, and executive vice dean of the USC Viterbi School of ...
A clutch stretch goes a long way
2024-02-02
Kyoto, Japan -- Cell biology has possibly never jumped to the next level in the same way.
In multicellular organisms, cell migration and mechanosensing are essential for cellular development and maintenance. These processes rely on talin, the key focal adhesion -- or FA -- protein, central in connecting adjacent cellular matrices and enabling force transmission between them.
Talins are commonly considered fully extended at FAs between actin filaments -- or F-actin -- and the anchor-like integrin receptor.
Yet, a research ...
Psychological care delivered over the phone is an effective way to combat loneliness and depression, according to a major new study
2024-02-02
The study, led by a team based at the University of York and Hull York Medical School and at Tees, Esk and Wear Valleys NHS Foundation Trust, found levels of depression reduced significantly and the benefits were greater than those seen for antidepressants.
Participants in the study reported their levels of emotional loneliness fell by 21% over a three-month period and the benefits remained after the phone calls had ceased, suggesting an enduring impact.
The Behavioural Activation in Social ...
Insulin prices in US are more than twice as high as in other wealthy nations
2024-02-02
The gross price of insulin in the U.S. is more than nine times higher than in 33 high-income comparison nations, according to a new RAND report.
Although the cost differences of insulin between the U.S. and other nations varied depending on the comparison country and the type of insulin, U.S. prices were always higher -- often five to 10 times higher -- than those in other countries. The new report updates findings from earlier RAND work about U.S. insulin prices.
After accounting for rebates and other discounts often offered by drug ...
My love language is peer-reviewed research
2024-02-02
Feb. 1, 2024, TORONTO – From the Five Love Languages to the concept of “Happy Wife, Happy Life,” popular culture is riddled with ideas of how sex and relationships are supposed to work, but does the science back these ideas up? According to Faculty of Health Assistant Professor and Research Chair in Relationships and Sexuality Amy Muise, the answer is frequently no.
Ahead of Valentine’s Day, Muise, also director of the Sexual Health and Relationship (SHaRe) Lab, can offer alternative theories that are supported by her research and other literature in the ...
Husker researchers using metabolic model to study temperature stress on corn
2024-02-02
A research team led by Nebraska scientists has built the largest-ever metabolic model of corn to study how temperature stress affects the plant and how a certain fungus can help alleviate the problem.
The research is an expansion of earlier work with a metabolic model of corn roots that the same team used to study the plant’s nitrogen-use efficiency under nitrogen stress conditions, said Rajib Saha, Richard L. and Carol S. McNeel associate professor of chemical and biomolecular engineering and principal investigator. Saha and ...
New prescription drugs typically sold first in US
2024-02-01
Most new prescription drugs are sold first in the U.S. before they reach other nations, but ultimately important medications are sold across most wealthy nations within about a year of first sale, according to a new RAND report.
Researchers say the study’s findings have implications for the debate over whether efforts to reduce high prescription costs in the U.S. could hurt patients’ access to the newest drug treatments.
“Other wealthy nations -- all of which have much lower drug prices compared ...
LAST 30 PRESS RELEASES:
Study showcases resilience and rapid growth of “living rocks”
Naval Research Lab diver earns Office of Naval Research 2025 Sailor of the Year
New Mayo-led study establishes practical definition for rapidly progressive dementia
Fossil fuel industry’s “climate false solutions” reinforce its power and aggravate environmental injustice
Researchers reveal bias in a widely used measure of algorithm performance
Alcohol causes cancer. A study from IOCB Prague confirms damage to DNA and shows how cells defend against it
Hidden viruses in wastewater treatment may shape public health risks, study finds
Unlock the power of nature: how biomass can transform climate mitigation
Biochar reshapes hidden soil microbes that capture carbon dioxide in farmland
Reducing saturated fat intake shows mortality benefit, but only in high-risk individuals
Manta rays create mobile ecosystems, study finds
Study: Mixed results in using lipoic acid to treat progressive multiple sclerosis
Norbert Holtkamp appointed director of Fermi National Accelerator Laboratory
New agentic AI platform accelerates advanced optics design
Biologists discover neurons use physical signals — not electricity — to stabilize communication
Researchers discover that a hormone can access the brain by hitchhiking
University of Oklahoma researcher awarded funding to pursue AI-powered material design
Exploring how the visual system recovers following injury
Support for parents with infants at pediatric check-ups leads to better reading and math skills in elementary school
Kids’ behavioral health is a growing share of family health costs
Day & night: Cancer disrupts the brain’s natural rhythm
COVID-19 vaccination significantly reduces risk to pregnant women and baby
The role of vaccination in maternal and perinatal outcomes associated with COVID-19 in pregnancy
Mayo Clinic smartwatch system helps parents shorten and defuse children's severe tantrums early
Behavioral health spending spikes to 40% of all children’s health expenditures, nearly doubling in a decade
Digital cognitive behavioral treatment for generalized anxiety disorder
Expenditures for pediatric behavioral health care over time and estimated family financial burden
Air conditioning in nursing homes and mortality during extreme heat
The Alps to lose a record number of glaciers in the next decade
What makes a good proton conductor?
[Press-News.org] New molecule from University step closer to treatment for rare diseaseA molecule created at the University of Auckland is one step closer to becoming a treatment for an extremely rare and severely debilitating neurological disorder called Phelan-McDermid syndrome.