Saturday, March 6, 2021
Home Life Style Health & Fitness New Experimental Drug likely to have potential to Improve quality of life...

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

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.

NewsGram brings to you current foreign news from all over the world.

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.

NewsGram brings to you top news around the world today.

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.

Check out NewsGram for latest international news updates.

“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)

STAY CONNECTED

19,120FansLike
362FollowersFollow
1,773FollowersFollow

Most Popular

A Gentleman’s Guide With A Detailed Regime For The Amateur To The Pro

While picking the right products can be a daunting task, men are serious about grooming, self-care, and wellness routines. With the changes the world...

Indian Women Feel The Pressure To Seek Permission For Their Professional Choices

Do young Indian women still feel the pressure to seek permission for their professional choices? According to new OkCupid data, an overwhelming 79 percent...

Study: The Market For Luxury Smart Homes And Villas Is Constantly Thriving

As the real estate sector emerges out of pandemic, the market for luxury smart homes and villas in India is thriving and demand has...

Benefits of “Heeng” : The Extraordinary Spice of Every Indian Household!

In every Indian household, the most important kitchen ingredient that is never missed in every Indian household is "Heeng" or Asafoetida. It has a...

Can AI Get Common Sense? Facebook Model Shows The Way

In an advance to building machines with common sense, Facebook researchers have developed a new Artificial Intelligence (AI) model that can learn from any...

WHO: Ban On Vaping Can Be Counterproductive

Millions of vapers across Asia could feel forced to return to smoking if a proposal from the World Health Organization (WHO) on e-cigarettes and...

Study: Smoking Cessation Drug Cytisine May Help Treat Parkinson’s Disease

A smoking cessation drug -- cytisine -- may help treat Parkinson's disease or stop its progression in women, say, researchers, including an Indian-origin. The...

5 Healthy Ingredients To Include In Our Diet To Channelize Our Inner Boss Lady

Women have always been the epicenter of our family lives, and are now budding into entrepreneurs, impactful team players, and opinion leaders that shape...

Recent Comments