What Is Spinal Muscular Atrophy?

Answer 1

Spinal muscular atrophy (SMA) most often affects babies and children and makes it hard for them to use their muscles. When your child has SMA, there's a breakdown of the nerve cells in the brain and spinal cord. The brain stops sending messages that control muscle movement. When that happens, your child's muscles get weak and shrink, and he can have trouble controlling head movement, sitting without help, and even walking. In some cases he can have trouble swallowing and breathing as the disease progresses. There are different types of SMA, and how serious it is depends on which type your child has. There's no cure, but treatments can improve some symptoms and in some cases help your child live longer. Researchers are working to find new ways to fight the disease. Keep in mind that every child or adult who has SMA will have a different experience. No matter how much your child's movement is limited, the disease doesn't affect his intelligence in any way. He will still be able to make friends and socialize. Keyword: spinal muscular atrophy

Answer 2

FDA has approved the medicine for spinal muscular atrophy, the medicine is a gene therapy and it targets a defective gene that weakens a child's muscle so dramatically that they become unable to move, and eventually unable to swallow or breathe. It strikes 400 babies in the U.S. every year.

The new medicine, from Swiss drugmaker Novartis, is said to help infants that usually die of the disease by age 2.

The disease is caused by a faulty gene, which prevents the body to produce a protein necessary for healthy nerve cells. The drug works by supplying a healthy copy of the faulty gene, to allow the nerver cells to produce the protein.

However, this is the most expensive medicine ever. The treatment is priced at $2.125 million. Out-of-pocket costs for patients will vary based on insurance coverage.

Novartis said it will let insurers make payments over five years, at $425,000 per year, and will give partial rebates if the treatment doesn't work.

Comments

Hopefully that is a typo and the drug will cure, not kill all those with the most common form of the disease.

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I though the same thing.

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I was honestly concerned about that myself

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It's a typo, I'm so sorry I don't know what I was thinking then. Okay it's fixed now. Thanks for your feedback.