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New Experimental Drug likely to have potential to Improve quality of life for Infants suffering from a rare Muscle Disease

Spinal muscular atrophy is a genetic disease that affects around one in every 11,000 births

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Representational image. Pixabay

New York, December 7, 2016: A new experimental drug may have the potential to improve the quality of life for infants suffering from a rare, lethal neuromuscular disorder, US researchers have found.

Spinal muscular atrophy is a genetic disease that affects around one in every 11,000 births.

It affects the nerve cells in the spinal cord that connect to the muscles, causing them to waste away resulting in progressive muscle weakness and difficulty in breathing and eating.

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Infantile-onset, which is the most severe form of the disease, occurs from a defect in the gene called SMN2, responsible for producing survival motor neuron (SMN) — a protein critical for normal cell function.

It affects babies under the age of six months. Less than a quarter of those diagnosed with the disease will live up to two years without major feeding and breathing support.

The study — a phase 2 trial involving 20 babies with infantile-onset SMA — showed the treatment with the drug nusinersen could increase the production of SMN protein by modifying a closely-related gene to compensate for the genetic defect.

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Apart from being safe for use in babies as young as five-week-old, nusinersen was also found to halt progression of the disease and in many cases improve motor function.

In addition, nusinersen sometimes enabled children to gain skills such as sitting, rolling over, and standing — usually not seen in SMA Type 1 — as well as improved survival without depending upon the continuous use of a ventilator, the researchers explained.

“With nusinersen, these infants are not only living longer, but living better,” said lead author Richard S. Finkel from Nemours Children’s Hospital in Florida, US.

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“SMA is no longer a death sentence for infants. This treatment is by no means a cure, but it is more than we’ve ever been able to offer these families before,” Finkel added, in the paper published in The Lancet. (IANS)

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Early Exposure of Infants To Household Cleaning Products Can Make Them Prone To Asthma

Reading labels on cleaning products and choosing those that are not sprayed or contain volatile organic compounds will help minimise a child's exposure and balance the risk associated with using cleaning products in an effort to achieve a mould-free, low-allergen home

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The researchers hypothesize that chemicals in cleaning products may damage the respiratory lining by triggering inflammatory pathways of the immune system, leading to asthma and wheeze in Babies. Pixabay

Early exposure of babies to household cleaning products is associated with the development of childhood asthma and wheeze by age 3 years, a new study suggests.

“Our study looked at infants, who typically spend 80 per cent-90 per cent of their time indoors and are especially vulnerable to chemical exposures through the lungs and skin due to their higher respiration rates and regular contact with household surfaces,” said study lead researcher Tim Takaro from Simon Fraser University in Canada.

For the findings, published in the journal CMAJ, researchers looked at data from questionnaires completed by parents of 2022 children in the Canadian Healthy Infant Longitudinal Development (CHILD) Cohort Study who were exposed to cleaning products from birth to age 3-4 months. Participants in the CHILD Cohort Study were recruited from mostly urban centres in 4 provinces: Vancouver, BC; Edmonton, Alberta; Winnipeg, Morden and Winkler, Manitoba; and Toronto, Ontario.

The children were then assessed at age 3 years to determine whether they had asthma, recurrent wheeze or atopy (allergic sensitisation). The most common cleaning products used were hand dishwashing soap, dishwasher detergent, multisurface cleaners, glass cleaners and laundry soap.

The researchers found an association between early exposure to cleaning products and risk of asthma and wheeze. According to the study, scented and sprayed cleaning products were associated with the highest risk of respiratory issues.

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Early exposure of babies to household cleaning products is associated with the development of childhood asthma and wheeze by age 3 years, a new study suggests. Pixabay

The researchers hypothesize that chemicals in cleaning products may damage the respiratory lining by triggering inflammatory pathways of the immune system, leading to asthma and wheeze.

The modulation of the infant’s microbiome may also play a role, the study said. “These findings add to our understanding of how early life exposures are associated with the development of allergic airway disease, and identify household cleaning behaviours as a potential area for intervention,” said study lead author Jaclyn Parks.

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Reading labels on cleaning products and choosing those that are not sprayed or contain volatile organic compounds will help minimise a child’s exposure and balance the risk associated with using cleaning products in an effort to achieve a mould-free, low-allergen home, the study said. (IANS)