What is Spinal Muscular Atrophy
SMA is a rare genetic disease.
It affects a person’s motor neurons at a progressive rate.
This means that over time, it weakens the muscles involved with walking, breathing, swallowing, and all other voluntary movements.
The Basics
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.
The Diagnosis
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.
The Types
Three treatments are currently on the market, and many more are in the pipeline.
Spinraza and Evrysdi are meant to stop the progression of SMA, though it is not guaranteed.
Zolgensma, a gene therapy, is predicted to cure the disease. However, this drug is only safe for infants.