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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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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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Feeding Probiotics to Infants Daily May Reduce Antibiotic Prescription in Future

For the study, the team pooled data from twelve studies together. The probiotics used in the reviewed studies were strains of Lactobacillus and Bifidobacterium

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Can probiotic use help reduce antibiotic prescriptions in children? Find it out here.

Feeding probiotics to infants and children daily may significantly stave off the need for antibiotic treatment, a finding that may help address the global rise in drug-resistant infections, said researchers.

The study found that infants and children were 29 per cent less likely to have been prescribed antibiotics if they received probiotics as a daily health supplement.

The results, published in the European Journal of Public Health, are very intriguing, the researchers said.

“Given this finding, potentially one way to reduce the use of antibiotics is to use probiotics on a regular basis,” said Daniel Merenstein, professor at Georgetown University.

According to the World Health Organization (WHO), antibiotic resistance occurred among 500,000 people with suspected bacterial infections across 22 countries.

Reducing the use of antibiotics is one strategy in combating resistance.

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Say no to your kids for junk food, instead add healthy snacks. Pixabay

“We already have evidence that consuming probiotics reduces the incidence, duration, and severity of certain types of common acute respiratory and gastrointestinal infections,” Merenstein said.

However, it is not clear how probiotics help fight infections.

Merenstein said: “There are many potential mechanisms, such as probiotic production of pathogen inhibitors, immune regulation, among others.

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“We don’t know all the mechanisms probiotic strains may leverage. But since most of the human immune system is found in the gastrointestinal tract, ingesting healthy bacteria may competitively exclude bacterial pathogens linked to gut infections and may prime the immune system to fight others,” he explained.

For the study, the team pooled data from twelve studies together. The probiotics used in the reviewed studies were strains of Lactobacillus and Bifidobacterium. (IANS)