Experimental drug offers hope to 3-year-old Long Island girl with rare disease

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Kristin Thorne has the story.

An inspiring medical breakthrough is helping a 3-year-old girl who doctors didn't think would ever be able to walk.

The little girl has spinal muscular atrophy, and her success story thanks to an experimental drug may now lead to others.

"At 13 months, we started noticing things, changes in her," mom Dianne Larson said. "Her crawling slowed. All of a sudden, her feet weren't moving."

Dianne and Matthew Larson knew something was wrong, so they took their daughter Emma to a doctor. She was diagnosed with the disease, which is often fatal and leaves children unable to walk and sometimes even sit up or use their arms.

"When your child is diagnosed with something so terrible, you just go to the ends of the earth to see what is out there what we can do," Dianne said.

So they enrolled in a clinical trial that involved a new drug created and researched for years at Cold Spring Harbor Lab called nusinersen.

"Within three months of an injection, we saw an improvement in her," Dianne said.

And that has continued ever since.

"She's gained so much strength, doing things she's never done," Matthew said. "The progression of the disease stopped, and she's making gains every day."

Dr. Adrian Krainer led the team of researchers.

"Now it's in the hands of the FDA," he said. "They have to look at all the data to make sure they see that the drug is effective and safe."

The FDA has recently fast tracked the drug, which means it could be available within the next six months

The Larsons pray they continue to see improvements in not only Emma, but any child who will hopefully soon have access to what some researchers are calling a miracle drug.
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