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Family fights for research on rare genetic disease

Stem cell researcher Tadashi Sato holds a dish containing stem cell growth medium, at the University of Nebraska Medical Center, in Omaha, Neb., Monday, March 9, 2009. (AP Photo / Nati Harnik)
Stem cell researcher Tadashi Sato holds a dish containing stem cell growth medium, at the University of Nebraska Medical Center, in Omaha, Neb., Monday, March 9, 2009. (AP Photo / Nati Harnik)

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Date: Monday Sep. 6, 2010 5:19 PM ET

An Edmonton family is pushing for the government to fund more gene therapy and stem cell research, in the hopes it will bring a cure for the genetic disease affecting their young daughter.

Lisa Lohin says her two-year-old child, Enna, was diagnosed with an aggressive form of spinal muscular dystrophy shortly after birth.

The condition takes away the ability to move muscles on command, and Enna must be on a ventilator 24 hours a day.

"The doctors told us we had only months with her and there was nothing we could do -- there is no cure," Lohin told CTV News in Edmonton on Saturday. The toddler has already survived longer than anyone expected.

Lohin wants the government to put more money into research that could offer a cure for the disease, which affects one in 6,000 Canadian babies. One in 40 adults carry the gene.

"Gene therapy and stem cells is the big key for these children," she says. "Not only for Enna, but for a lot of other related genetic disorders."

Enna's pediatrician says research could eventually provide relief for families dealing with the debilitating disease.

"I think the possibility exists that we may not have to deal with it the way we do now at some stage," says Dr. Lyle McGonigle.

Lohin says she knows her daughter may not benefit from the research her family is fighting for. But every day Enna lives brings hope to other families coping with a similar diagnosis.

"She may not be able to sit up or run…but she's met a lot of milestones that we never thought we would ever see."

With files from CTV Edmonton's Dez Melenka

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