Evidence Suggests The Spinal Muscular Atrophy Gene Therapy Could Help all Patients Regardless of Severity

According to a story from BNN Bloomberg, data from three studies suggest that Zolgensma, a gene therapy for spinal muscular atrophy developed by Novartis, could be useful to all patients with the disease regardless of the severity. While the therapy is primarily intended for patients with the classic, most severe disease type, which can be lethal in as little as few months, patients with milder disease definitely stand to benefit from its approval.

About Spinal Muscular Atrophy (SMA)

Spinal muscular atrophy is a type of neuromuscular disorder in which the motor neurons are destroyed, leading to muscle wasting. In many cases, the disease is lethal. This disorder is linked to genetic defects of the SMN1 gene. This gene encodes a protein called SMN, and when not present in certain amounts, neurons are unable to function. There are different kinds of spinal muscular atrophy that are categorized by when symptoms first appear. These symptoms may include loss of reflexes, muscle weakness and poor muscle tone, problems with feeding and swallowing, developmental delays, respiratory muscle weakness, tongue twitching, and a bell shaped torso. The most effective treatment currently available for the disease is called Spinraza. To learn more about spinal muscular atrophy, click here.

Making a Difference

A single dose of Zolgensma has the potential to halt the process of neuron death in as little as a few weeks. This treatment is not only capable of stopping the progression of spinal muscular atrophy, but also allowing for the resumption of normal development and movement ability. Still, prompt treatment is essential, as neuron death is impossible to reverse.

Zolgensma also offers advantages over Spinraza, which is currently the only approved therapy for spinal muscular atrophy. Spinraza is effective at controlling the disease, even in the milder forms, but it must be administered several times per year. While it is unclear how the prices will compare, Zolgensma only required a single administration for long term benefit.

At this juncture, there is no scientific basis that would support the use of the two therapies in combination.

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