What is life expectancy after Zolgensma?

The one-off gene therapy treats SMA, a rare and often fatal genetic disease that causes paralysis, muscle weakness and progressive loss of movement. Babies born with severe type 1 SMA – the most common form of the condition – have a life expectancy of just two years.

Does Zolgensma increase life expectancy?

The latest data suggests that Zolgensma can provide rapid and sustained improvement in motor function for young children with type 1 SMA and prolong their lives.

What is the life expectancy of a child with SMA?

SMA Type 1 is a life-limiting condition. Though it is not possible to accurately predict, for the majority of children (approximately 95\%) life expectancy is less than 18 months unless pharmacological treatment is introduced.

Do kids with SMA survive?

Life expectancy Most children with type 1 SMA will only live a few years. However, people who’ve been treated with new SMA drugs have seen promising improvements in their quality of life — and life expectancy. Children with other types of SMA can survive long into adulthood and live healthy, fulfilling lives.

What does Zolgensma do for SMA?

Zolgensma is an SMN-enhancing therapy that works by replacing the function of the missing or nonworking SMN1 gene with a new, working copy of an SMN gene. A virus, AAV9, carries the replacement gene into the body. This virus “infects” the cells with the new DNA. As with all treatments, timing is critical.

How often is Zolgensma given?

Zolgensma is given as a one-time dose. Clinical studies haven’t looked at how effective the drug may be if a child receives more than one dose. Zolgensma hasn’t been studied as a treatment for advanced SMA. “Advanced” means the disease has worsened.

What does Zolgensma do?

Zolgensma is an SMN-enhancing therapy that works by replacing the function of the missing or nonworking SMN1 gene with a new, working copy of an SMN gene. A virus, AAV9, carries the replacement gene into the body. This virus “infects” the cells with the new DNA.

How quickly does Zolgensma work?

How long does it take to work? Zolgensma starts working to treat SMA as soon as it’s injected. In clinical studies of the drug, some children saw improvements in their SMA symptoms as early as 1 month after they received the drug.

Is SMA type 2 curable?

There’s currently no cure for SMA, but the Food and Drug Administration (FDA) has recently approved several new treatments for both type 1 and type 2 SMA, including innovative gene therapies, with many more potential treatments on the horizon.

Does Zolgensma work well?

Clinical studies have shown Zolgensma to be effective for treating SMA caused by genetic mutations* in the SMN1 gene. The drug works by replacing an abnormal SMN1 gene with a normal SMN1 gene. However, Zolgensma won’t reverse any effects of SMA that happened before your child received the drug.

How effective is Zolgensma for infants with SMA?

In two remarkable single arm clinical trials, infants who had already developed symptomatic type 1 SMA and then received Zolgensma not only survived well past their predicted date of death, but experienced improvement in neuromuscular function. Zolgensma is approved for infants under the age of 2 with any type of SMA, whether symptomatic or not.

What is the average life expectancy of someone with SMA?

The mean age was 4.3 years (range 2.86.1 years) and the mean time since gene therapy treatment was 2.4 years (range 1.8–2.8 years). Four patients (50 percent) were not receiving concomitant disease-modifying SMA therapy.

What is the long-term prognosis of Zolgensma?

Zolgensma led to achievement of new milestones years after treatment in two long-term follow-up studies and resulted in sustained durability in children with SMA now up to six years old and more than five years post-treatment

What is Zolgensma used to treat?

Zolgensma is indicated for the treatment of children less than two years of age with SMA. The product is an adeno-associated virus vector-based gene therapy that targets the cause of SMA.