Spinal muscular atrophy (LOVE) is a rare genetic and degenerative disease, affecting 65 per 100,000 people. Its main feature is the body’s inability to produce a protein necessary for the development and survival of motor neurons, which are part of the body’s vital voluntary movement processes, such as swallowing, breathing and moving.
There are degrees of SMA, which vary according to the age at which the first symptoms appear and the individual’s level of impairment.
Unfortunately, there is no cure for this disease and, in cases of early diagnosis, which occurs before the age of two, there is a possibility of genetic treatment, which stops the progress of the disease and helps the body to produce the protein necessary for good health. motor neuron performance.
Most Expensive Medicine in the World
The drug in question is called Zolgensmaapplied in a single intravenous dose, but which, in order to take effect, must be used before the child turns two years of age.
The problem is that Zolgensma is the most expensive drug in the world and, as it is sold in dollars, it costs more than BRL 10 million.
The use of this drug is essential for the treatment of type 1 of the disease, which is the most serious and fatal in 90% of cases. That is why many families turn to social networks to carry out fundraising campaigns and, in addition to the exorbitant amount, these people have to race against time, since, after the age of two, the drug would have its effect greatly reduced and nor is it often applied.
The value of the drug has to do with the degree of complexity for its development. For being the first in the world to use the gene therapyZolgensma is a great achievement for Science, being able to repair the defective genes that cause the disease.
Furthermore, the company Novartis, which produces the drug, is the only one that develops this type of drug and also has the patent for the product, being able to determine its price without fear of competition.
In Brazil, the SUS does not provide the medication, but some families go to court and manage to have the costs covered by the State. As not everyone is so lucky and can’t wait too long, the solution is to do crowdfunding and fundraising campaigns on the internet.
Now you know: when you find any publication about AME and Zolgensma, help by contributing or sharing, after all, as it is common to hear from families who live this reality: those who have AME, are in a hurry.