DALLAS - She may be in a wheelchair, but 14-year-old Lanie Hannah doesn't let that stop her from having fun.
She has SMA. It attacks the body's motor neurons and causes paralysis.
"It affects my daily life, like not being able to do certain things," she said.
"Put her in bed, take her out of bed, dressing. I mean, it affects everything that she does," said Lana Hannah, Lanie's mom.
There is no cure for SMA, but 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.
The gene therapy is injected into the spinal fluid. The idea is the drug will enter into the nerve cells and change the protein production so that the normal SMN gene is created. It could mean fewer symptoms and more strength for patients.
"Our hope, absolutely, is that someday these children will not experience the symptoms," Iannaccone said.
Lanie was in the phase I trial. She hopes the therapy will be the answer she's been waiting for because she has big plans for the future.
"I want to go to college. I want to be a teacher, and I want to teach English," Lanie said.
"Everything that she does amazes me," Lana Hannah said.
Lanie has a milder form of the disease, but she has never been able to walk. Researchers said the next step is to study more patients in a phase II trial to see if the gene therapy does what they hope it will.
Allentown, PA 18102