Tuesday November 19, 2019

Drug that Helps Regulate Bone Development to Boost Growth Rates of Children with Dwarfism on Global Trial

The drug, vosoritide, was generally well tolerated by patients

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BP drug, bowel
BP drug may up risk of bowel condition. Pixabay

 A drug that helps regulate bone development has been found to boost growth rates in children with achondroplasia, the most common type of dwarfism, in a global trial.

The patients’ average boost in height to about 6 cm (2.4 inches) per year was close to growth rates among children of average stature, and the side effects of the drug were mostly mild, said study co-author Julie Hoover, Associate Professor at the McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University.

“Right now, the results of the study show an impact on growth, and this effect is sustained, at least over nearly four years in this trial,” Hoover said.

Results of the phase-2 trial published in the New England Journal of Medicine showed that the drug, vosoritide, was generally well tolerated by patients.

Drug, Children, Dwarfism
A drug that helps regulate bone development has been found to boost growth rates in children. Pixabay

On average, participants in the trial grew at a 50 per cent faster compared to baseline with no adverse effects on body proportion, the results showed.

Achondroplasia is caused by over-activity of a signal that stops growth, and could be likened to overwatering a plant, said lead author Ravi Savarirayan, Professor at Melbourne’s Murdoch Children’s Research Institute in Australia.

“This drug basically kinks the hose so that the plant gets the right amount of water and can resume regular growth,” Savarirayan said.

Achondroplasia is a genetic bone disorder affecting about one in every 25,000 infants.

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It is caused by a mutation in the FGFR3 gene that impairs the growth of bones in the limbs, the spine, and base of the skull.

The most common health complications experienced by children with achondroplasia are spinal cord compression, spinal curvature and bowed legs. About half of these children will need spinal or other surgery.

Unlike other treatments – such as growth hormone and limb-lengthening surgery – that focus on symptoms, vosoritide focuses on the underlying cause of achondroplasia and directly counteracts the effect of the mutation that slows growth.

Drug, Children, Dwarfism
The patients’ average boost in height to about 6 cm (2.4 inches) per year was close to growth rates among children of average stature. Pixabay

The study ran over four years across research centres in Australia, France, Britain and the US with 35 children assigned to one of four groups receiving daily subcutaneous doses of the drug in increasing amounts.

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The results showed vosoritide demonstrated dose-dependent increases in centimetres grown per year during the first six months, with improvements maintained over the study extension period of a further three years. (IANS)

Next Story

Study Says, Early Signs of Diabetes Can be Observed in Children

The study tracked over 4,000 participants of the Children of the 90s study, a birth cohort established in Bristol in the early 1990s

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Diabetes
The research was conducted among young healthy people who were generally free of type 2 Diabetes and other chronic diseases to see how early in life the effects of diabetes susceptibility become visible. Pixabay

Researchers have found that early signs of adulthood type 2 Diabetes can be seen in children as young as 8 years old.

Type 2 diabetes is most often diagnosed in middle age or later, with its symptoms slowly developing over many years.

“It’s remarkable that we can see signs of adult diabetes in the blood from such a young age, this is about 50 years before it’s commonly diagnosed.

“This is not a clinical study; nearly all participants were free of diabetes and most will not go on to develop it. This is about liability to disease and how genetics can tell us something about how the disease develops,” said study researcher Joshua Bell from the University of Bristol in the UK.

The research was conducted among young healthy people who were generally free of type 2 diabetes and other chronic diseases to see how early in life the effects of diabetes susceptibility become visible.

The study tracked over 4,000 participants of the Children of the 90s study, a birth cohort established in Bristol in the early 1990s.

The researchers combined genetics with an approach called ‘metabolomics’, which involves measuring many small molecules in a blood sample to try and identify patterns that are unique to type 2 diabetes.

According to the findings, the research team analysed 162 pieces of genetic information and combined this with 200 measures of many small molecules in a blood sample, known as metabolics, to identify signs of type 2 diabetes.

Diabetes
Researchers have found that early signs of adulthood type 2 Diabetes can be seen in children as young as 8 years old. Pixabay

Data was taken once in childhood — at 8 years old, twice in adolescence aged 16 and 18 years and once in young adulthood aged 25 years.

They found levels of HDL cholesterol were reduced at age 8, while inflammatory glycoprotein acetyls and amino acids were elevated in 16 and 18 year old teenagers.

These metabolic features could be targeted to prevent young people from going on to develop type 2 diabetes in the future, the researchers said.

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The findings were presented at the European Association for the Study of Diabetes (EASD) Annual Meeting in Barcelona. (IANS)