What is Spinal Muscular Atrophy

  • 1 in 40 people carry the gene that causes SMA.

  • This could be someone in the office, grocery store, or even your family.

  • Both parents must carry the gene to pass the disease to their child.

  • There is a 25% chance the child will be affected. A blood test will determine this.

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The Diagnosis

  • Two treatments are currently on the market with many more in the pipeline.

  • The two treatments are Spinraza and Zolgensma.

  • Spinraza is meant to stop the progression of SMA.

  • Zolgensma, a gene therapy, is predicted to cure the disease. However, this drug is only safe for infants.

  • SMA is a rare, genetic disease.

  • It affects a person’s motor neurons at a progressive rate.

  • This means it weakens the muscles involved with walking, breathing, swallowing, and all other voluntary movements over time.

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The Basics

  • There are 4 types of SMA— I, II, III, or adult onset.

  • Type I is the most severe and typically diagnosed shortly after birth.

  • Types of SMA are classified by milestones reached as an infant or child.

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The Types

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The Treatments

Treatments serve as a great reminder we are headed in the right direction. But with a disease as aggressive as SMA, the battle isn’t over just yet.

For a comprehensive analysis of SMA, please visit SMA News Today.