![New Gene Therapy Medication can Treat Muscle Disease in Children New Gene Therapy Medication can Treat Muscle Disease in Children](https://images.medindia.net/health-images/1200_1000/new-gene-therapy-shows-promise.jpg)
In the earlier study, published in NEJM in November, Mar Tulinius also was responsible for the Swedish part. Then Spinraza was administered for the most severe form of spinal muscular atrophy, SMA type 1, which afflicts children before they are six months old.
This time the study concerned SMA type 2, in which the illness occurs when children have learned to sit by themselves but have not yet begun to walk. The muscles gradually weaken, the children never stand upright on their legs and ultimately they need respirators to survive.
Two out three children in the current study received Spinraza; one out of three received a placebo. And this time, too, it turned out that the drug with the active substance nusinersen, which partly compensates for an inherited genetic error, altered the children's condition.
Their ability to sit up straight, raise their hands, move by rolling around, etc., was improved by an average of four points on the scale used to measure changes in spinal muscular atrophy. At the same time, children in the placebo group regressed an average of 1.9 points on the scale.
"The natural progression is for the children to lose points on this scale. Although there are variations within the group, they are never destined to get better. The fact that strength and mobility increased in those who received the drug is really quite amazing," Mar Tulinius said.
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Every year 10 or so children in Sweden are diagnosed with SMA. The two most difficult variations, type 1 and 2, are covered by a recently issued recommendation from the Swedish Council for Novel Therapies (NT-rådet) of Sweden's municipalities and county councils that under certain conditions and strict control, children should be offered treatment with Spinraza.
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Source-Eurekalert