Good News / Bad News for Sufferers of Spinal Muscular Atrophy

On Friday the FDA approved a new gene therapy drug, Zolgensma, “a one-time treatment designed to help young children with spinal muscular atrophy.”

“Around 10,000 to 25,000 U.S. children and adults are estimated to have the disease, which occurs in one of every 6,000 to 10,000 births. Patients rarely live into adulthood.”

Because a dose of the drug has to be genetically tailored for each individual patient, Zolgemsa costs $2.1 million per  treatment, making it the most expensive drug in the world.

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3 Comments on Good News / Bad News for Sufferers of Spinal Muscular Atrophy

  1. Just declare yourself an illegal alien, you will get whatever you need. Money is no object, it grows on trees in the land of milk and honey.

    9
  2. Make everything ‘a right.’
    Implement Green New Deal.
    Medicare for all!
    Universal Socialism.
    Print unlimited money.
    50%+ unemployment
    Provide for over 50% of the population.
    Runaway inflation.
    Bankrupt the country.
    Go back to the stone age – and start over.

    5
  3. This is a case where community churches and organizations used to step in and raise money for a child in need. It can still happen.

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