Known as a rare, genetic disorder,

Spinal muscular atrophy (SMA) is a progressive neuromuscular disease that affects a person’s motor neurons. Motor neurons are the little messages that fire to the muscles, telling them what to do. So, with SMA, these messages get lost in translation, ultimately resulting in the inability to move and progressive muscle weakness.

 
 
 

Known as a rare, genetic disorder,

Spinal muscular atrophy (SMA) is a progressive neuromuscular disease that affects a person’s motor neurons. Motor neurons are the little messages that fire to the muscles, telling them what to do. So, with SMA, these messages get lost in translation, ultimately resulting in the inability to move and progressive muscle weakness.

FAQ

 

Am I a carrier?

1 in 40 people carry the gene that causes SMA. This means someone in your grocery store, business department, or extended family could be a carrier. In order to pass the gene onto a child, both parents must be carriers. There is a 50% chance your child will become a carrier and a 25% chance your child will have SMA.

What are the symptoms of SMA?

There are four different types of SMA (I, II, III, IV, or adult-onset), and each type has its own list of symptoms and levels of severity. Those living with type I have the most severe form of the disease and often require palliative care as part of their daily treatment regimen. While each diagnosis is classified by type, SMA presents itself in many different ways.

How is it diagnosed?

A simple blood test can determine if you or your loved one has SMA. In newborns, the failure to reach important milestones such as rolling, crawling, and walking typically elude to the early onset of the disease. There are 5 types of SMA, and each type is determined by how many physical milestones are reached as a child.

Are there any treatments?

Yes! Currently, there are 3 treatments on the market: Spinraza, Evrysdi, and Zolgensma. Access to these treatments is limited to many pending on their insurance and location. While these treatments aren’t a cure, they are rewriting the future of SMA, and many other treatments in the pipeline are promised to do the same.

 
 

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