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Spinal Muscular Atrophy (SMA) is a rare genetic disorder that leads to the progressive loss of motor neurons, resulting in muscle weakness and atrophy. It is one of the leading genetic causes of infant mortality, affecting approximately 1 in 10,000 live births. Traditional treatments have been limited, but recent advancements in gene therapy have brought new hope to families worldwide.
One such breakthrough is Zolgensma (onasemnogene abeparvovec-xioi), a one-time gene therapy that addresses the root cause of SMA by delivering a functional copy of the SMN1 gene. This therapy has revolutionized the treatment landscape for SMA, offering potential life-saving benefits for affected children.
Zolgensma is an adeno-associated virus (AAV) vector-based gene therapy designed to treat the genetic root cause of SMA. It delivers a functional copy of the SMN1 gene to motor neurons, enabling the production of the SMN protein essential for motor neuron survival and function.
SMA is caused by mutations in the SMN1 gene, leading to insufficient levels of the SMN protein. Zolgensma utilizes a non-replicating AAV9 vector to transport a functional SMN1 gene into motor neurons. Once inside the cell nucleus, the introduced gene produces the SMN protein, halting disease progression and preserving motor function.
Zolgensma is administered as a single intravenous infusion. Clinical studies have demonstrated its efficacy in improving motor function and achieving developmental milestones in infants with SMA. Early treatment, particularly before the onset of symptoms, has shown the most significant benefits.
Zolgensma is available through specialized treatment centers globally. However, its high cost has been a barrier for many families. Recognizing the need for broader access, World Scientific Impact offers Zolgensma at a reduced price of $637,500, aiming to make this life-saving therapy more accessible to those in need.
World Scientific Impact is committed to supporting humanitarian efforts. Proceeds from the sale of Zolgensma and other products contribute to initiatives aiding the less privileged, homeless, disabled individuals, and communities affected by war and natural disasters.
The mission of World Scientific Impact extends beyond commercial interests. By purchasing products like Zolgensma, customers contribute to the World Scientific Impact’s humanitarian projects. These initiatives focus on providing medical care, mobility aids, and essential resources to underserved populations worldwide.
In addition to Zolgensma, World Scientific Impact offers a range of products aimed at improving health and well-being:
Each purchase supports the foundation’s mission to aid those in need.
Zolgensma represents a significant advancement in the treatment of SMA, offering hope to families affected by this debilitating condition. Through the efforts of World Scientific Impact, access to this therapy is becoming more attainable, with proceeds supporting vital humanitarian initiatives.
For more information on Zolgensma and other products, visit the Biotech Products page.
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