Therapy hopes to relieve patients of SMA symptoms
Posted: April 23, 2014 at 5:47 am
SOUTH BEND, Ind.--- She may be in a wheelchair, but Lanie Hannah, 14, doesn't let that stop her from having fun.
Hannah has SMA which attacks the body's motor neurons and causes paralysis.
Spinal Muscular Atrophy or, SMA, occurs in almost one out of every 10,000 births.
It's the most common, fatal genetic disease in infants.
If patients are lucky enough to make it through infancy they are usually confined to a wheelchair.
"It affects me in my daily life, and it keeps me from being able to do certain things, said Lanie
"Putting her in bed, taking her out of bed, dressing, I mean it affects everything that she does, said Lana Hannah, Lanies mom.
There is no cure and there are no treatments; however, a new clinical trial is offering hope for the first time.
Doctors are studying an experimental therapy that targets more than just symptoms, it targets mutated SMN genes, which are responsible for SMA.
"With this treatment, we are targeting the disease," said Dr. Susan T. Lannaccone, a Pediatric Neurologist at the Childrens Medical Center in Dallas/UT Southwestern.
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Therapy hopes to relieve patients of SMA symptoms