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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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Children With Pets During Infancy Less Likely To Develop Allergies: Study

In another experiment, which included 249 children, it showed that the allergy rate for children growing up without a pet was 48 per cent

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Allergies
People raised in cities without pets at risk from mental illness. Pixabay

Children who live with pets when they are infants are less likely to develop allergies and other diseases later in childhood, a Swedish study found.

The study sought to learn about the possible benefits of germ exposure to infants living with pets in their home.

For the study, the researchers from the University of Gothenburg included 1,029 children who were either seven or eight years old.

In the first experiment, findings, published on the open access site, ‘PLOS ONE’, the researchers found that the incidence of allergies (which in this study included asthma, eczema, hay fever and allergic rhinoconjunctivitis) was 49 per cent for children who had not been exposed to pets as infants.

Allergies
The number fell to 43 per cent for children who had lived with a single pet as an infant and to 24 per cent for children who had lived with three pets. Pixabay

The number fell to 43 per cent for children who had lived with a single pet as an infant and to 24 per cent for children who had lived with three pets.

Also Read: Tips To Keep Pets Warm in Winter

In another experiment, which included 249 children, it showed that the allergy rate for children growing up without a pet was 48 per cent, 35 per cent for children with one pet and just 21 per cent for children who had grown up with multiple pets.

Taken together, the two datasets showed that the more exposure infants have to pets, the less likely they are to develop allergies later in life, the team concluded. (IANS)