Today, the US Federal Food and Drug Administration has issued a report on the study of a new type of gene therapy for children under two years of age, aimed at correcting the symptoms of SMA, the so-called spinal muscular atrophy. This is a very rare disease that is associated with genetic damage, in which the development of muscle nerves is so difficult that babies born with this disease rarely live to two years, primarily due to problems of breathing and swallowing. However, new genetic therapy has now been approved after testing it in clinical trials.
The therapy itself is called Zolgensma and is a manipulation of the modification of one specific gene called survival motor neuron 1 – this is the working title, but experts say that the correction of other genetic sites is also at the center of attention of the new therapy. According to preliminary clinical trials, this therapy has already helped most of the children up to two years old to survive this age, while significantly improving the neuromotor regulation of the spinal cord and its associated nerves.
It is worth noting the fact that the clinical trials themselves included work with 36 babies, 21 of which experienced relief during the course of the illness and even managed to survive the fatal statistical mark. This was connected with the issuance of a permit from representatives of the Federal Commission regarding the extension of this type of therapy to increasingly diverse medical practices – thus, it is already being used as part of new drugs and methods.
However, representatives of the Commission also stated that the use of this type of therapy may lead to some previously announced side effects – such as a deterioration of the liver or its complete failure, as well as a lack of production of certain enzymes. However, observing the current progress in improving the therapy, it can be said that the course of the research is going in the right direction.