One in 5,000 babies is born with SMA ( a terminal condition called spinal muscular atrophy ). Eighty percent of those babies have a severe form of the disease. No treatment has shown effect against this disorder and these babies rarely live until their second birthday . Although children with SMA have many physical disabilities, they do score well on IQ tests.
The pathology of SMA is similar to an adult disease called ALS or Lou Gehrig’s disease. The drug riluzole slows the progression of ALS, and a small study shows it may do the same for babies with SMA.Of the 10 babies in the trial, three received a placebo. All died in the first year. Seven were given riluzole. Four of those babies died too, but three survived and are still taking the drug. “We have three children still living. One at age 5. One at age 4, and one at age 3,” researchers say.
Emily Gallagher seemed healthy when she was born. Her mother, Nina Kenney, says, “At about 3 months was when we noticed that she wasn’t doing what she was supposed to do as far as gross motor development -- holding her head up, sitting up by herself.”At 9 months, researchers diagnosed Gallagher with severe spinal muscular atrophy, a disease that almost always causes respiratory failure and death by age 2. She is now 5 and has been on a ventilator since she was 10 months. She can’t swallow, so she’s fed through a tube, but she can and loves to talk.
Gallagher was not in the study, but she will start taking riluzole with the hope of extending her life even more say specialists. Her mom says, “Every day’s a miracle, so if it’s going to give us another day, that’s wonderful.”
