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Babies with spinal muscular atrophy receive “the most expensive drug in the world”

 


The three-month-old baby became one of the first patients in the United Kingdom to be treated with a potentially life-saving drug with the NHS, which can extend the lifespan of children with spinal muscular atrophy.

Iley Cadle-Birch, who was diagnosed with SMA type 1 at the age of 4 weeks, received one-time gene therapy at the Royal Children’s Hospital in Bristol on June 30.

Until two years ago, children with this condition had no treatment options. This is the leading cause of genetic death in adolescents.

However, babies can sit, crawl, or walk after being treated with the US gene therapy Zolgensma, which is called the world’s most expensive drug.

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Jade Cadle-Billingham first noticed something wrong when his son Riley was three weeks old (University Hospitals Bristol and Weston NHS Foundation Trust / PA).

The list price is £ 1.79 million each and is available on the NHS after the medical service signed a contract with the manufacturer Novartis Gene Therapies in March.

A one-hour intravenous infusion works by providing a functional copy of the gene known as SMN1. It requires the body to make proteins that are essential for the normal functioning of nerves that control muscle movement.

Patients with SMA have a defective copy of this gene that, when delivered by intravenous infusion, directs the body to translocate to nerve cells, produce proteins, and restore nerve function.

Babies born with the most common form, type 1 SMA, experience progressive weakness, lack of exercise, and dyspnea, with a life expectancy of 2 years.

Studies have shown that a single treatment with Zolgensuma helped babies with SMA sit, crawl, and walk, eliminating the need to wear a ventilator.

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Riley Cadle-Before being treated at the Bristol Royal Hospital for Children (University Hospitals Bristol and Weston NHS Foundation Trust / PA), I birched with my father Ryan.

The Weston NHS Foundation Trust (UHBW), which includes the University of Bristol Hospital and the Royal Children’s Hospital in Bristol, is one of only four centers nationwide that manages gene therapy.

Others include the University of Manchester NHS Foundation Trust, Sheffield Children’s NHS Foundation Trust, and Eberina London Children’s Hospital.

Riley from Gloucester received an injection of gene therapy last week after responding well to another treatment for SMA called Spinraza.

His mother, Jade Caddle Billingham, first noticed something was wrong with Riley’s hands, and the midwife also found some problems with his breathing.

He was taken to the hospital for a series of tests to check his reflexes and was later diagnosed with SMA type 1.

After a difficult start, including life support, Riley began receiving Spinraza.

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Riley Cadle-Birch received gene therapy in June after being diagnosed weeks after birth (University Hospitals Bristol and Weston NHS Foundation Trust / PA).

Ms. Cadle-Billingham said:

“When I started to suspect he had an SMA, I started investigating the condition and came across Zolgensma, which was only announced to have been approved by the NHS at the time, but where can I get it, Riley? I still didn’t know if I could have it.

“We want to do everything we can to raise awareness of this life-changing treatment and give hope to other SMA families experiencing the same.

“Getting this treatment is like giving him a future.”

Dr. Kayal Bijaya Kumar, a pediatric neurology consultant at Bristol Royal Hospital for Children, said:

“Data from scientific studies show that this treatment can significantly change the lives of children with spinal muscular atrophy.

“We are very pleased to be selected as one of the four national centers in the UK. We will work closely with other centers to ensure that eligible patients are treated wherever they live. I am looking forward to it.

“We hope that the success of this treatment will serve as a starting point for the availability of more gene therapies in the future.”

Sources

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2/ https://www.belfasttelegraph.co.uk/news/uk/baby-with-spinal-muscular-atrophy-receives-most-expensive-drug-in-the-world-40623587.html

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