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Spinal muscular atrophy treatment with gene replacement therapy

Spinal muscular atrophy treatment with gene replacement therapy
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Type of care




About the offer

Spinal muscular atrophy treatment with gene replacement therapy remains among the newest yet most promising methods that can help prevent any symptoms if started early and performed consistently. Nowadays, many techniques that can help replace a malfunctioning gene causing spinal muscular atrophy with a normal one undergo clinical trials, and few are already being approved. Right before the start of treatment, medical practitioners need to get as much information about the patient's health and disease as possible, so they prescribe various diagnostic procedures and lab tests. Onasemnogene abeparvovec-xioi (Zolgensma) helps to replace the malfunctioning SM1 gene with a normal one and uses the so-called scAAV9 delivery approach. In this drug, adequately functioning SM1 genes are associated with adenovirus that enters the nerve cells and helps to integrate normal genes into the nerve cells. As a result, the patient's motor neurons start to function correctly. Also, the treatment leads to the production of healthy nerve cells in the future. Zolgensma can be delivered via intravenous injections, injections in the spinal canal, or other methods, depending on the patient's age, current condition, and severity of symptoms. Gene replacement therapy is one of the best methods for kids, yet getting a neurologist's appointment is essential to ensure they can successfully enroll.

What’s included

Medical service

  • clinical history-taking
  • medical records review
  • physical examination
  • consultation with a neurologist
  • consultation with a genetic specialist
Laboratory tests
  • complete blood count (CBC)
  • biochemical analysis of blood (kidney and liver function tests)
  • inflammation blood tests
  • DNA test for SMN gene mutation (on the indication, price is not included)
  • neurological examination
  • electromyography
  • nerve conduction study
  • muscle biopsy (on the indication, price is not included)
  • histopathological specimen examination (on the indication, price is not included)
  • pre-procedure patient preparation
  • gene replacement therapy for SMA: onasemnogene abeparvovec-xioi (Zolgensma)
  • post-procedure care
  • symptomatic treatment
  • cost of essential medicines
  • cost of essential materials
  • nursing service
  • discharge medical records
  • further recommendations


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How long does the offer take?

The offer is designed for 7 days.

Does the offer require you to stay in the clinic overnight?

Where can I see the media files?

What are the age restrictions?

The offer is eligible for both adults and kids.

Can I customize the content of the offer?

Yes, the components of the deal can be changed. Get in touch with a vendor to create a personalized proposal.

Individual cost estimate. Non-binding 100% free assessment.

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