FDA approves first drug to treat spinal muscular atrophy

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Spinal muscular atrophy is a genetic, frequently fatal disease that causes severe weakness and muscle wasting.

It attacks motor neurons, so things like coughing and holding your head up, Max Lasko can’t do.

But, the Laskos like to focus on what Max can do, like play music, communicate through an emphasis on syllables or movement of the eyes, and bring joy to people.

Max’s list of “can do’s” is about to multiply, thanks to the very first drug, approved to treat individuals with SMA, called Spinraza.

“The best expectation and what we really hope for is this stops anything from getting worse from that point onwards, and that’s the chance to improve things over time,” said Kenneth Hobby, president of Cure SMA.  “What we’re doing is actually really going in and targeting the very first causes of the disease, rather than just the symptoms in the muscles.”

The FDA approved Spinraza months before the go-ahead was expected, but for the Laskos, who provide Max with round-the-clock care, this is a long-awaited moment.

“When we got the news, I looked at my wife and saw tears rolling down her cheeks,” said Jonathan Lasko, Montgomery County resident.  “But, it will take time to see how it helps and to what degree it helps him and many others, but we’re hopeful, we’re very hopeful.”

Max will receive his first injection in the fluid surrounding the spine on Feb. 7.

“Over the last 30 or so years, thousands of people involved, hundreds of events, and it all kind of comes together just in one moment; it’s quite amazing,” said Hobby.

Cure SMA is working to get this treatment fully covered by insurance and available across the country.

Copyright 2019 Nexstar Broadcasting, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.

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