Spinal muscular atrophy is a very dangerous disease, which condemns those who suffer from it to a reduced life expectancy. However, there is one drug that cures this ailment, but it has a high cost: Zolgensma.
The problem of raising money to receive Zolgensma treatment affects many families in different parts of the world. The ailment is not so common and at the same time it is quite difficult to fight, but the specialists of the American company AveXis found a cure. As of 2018, this company became part of the Swiss pharmaceutical giant Novartis, which now sells Zolgensma in different parts of the world for those who need it.
The disease is hereditary and affects the neuromuscular system. This ailment progresses rapidly and over time people who suffer from it lose the ability to stand, sit, move, swallow and breathe. However, intellectual capacities remain intact. Babies with SMA, as the disease is known, in most cases barely survive to two years of age.
The disease is caused by a mutation in the SMN1 gene that causes a reduction in the amount of the SMN protein, necessary for the survival of motor neurons. One of the benefits of the drug, once inside the body, is that it transports the SMN1 transgene to motor neurons and in this way causes an increase in the amount of the SMN protein. The drug has side effects, but they are negligible compared to the symptoms of SMA.
Side effects include nausea and increased liver enzymes.
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The price of the drug is around $2 million due to high development and production cost. Very few families or, to be more precise, hardly anyone can afford to buy a dose of this much-needed drug to save the life of their loved one. Parents of children with SMA try to get financial help through social media, but there is also another way to get the medicine.
What is the most expensive drug on the planet like?
Zolgensma is not simply the most expensive drug on the planet, it is a true breakthrough in gene therapy, a long-awaited invention for many families around the world. If it weren’t for its huge price tag, this drug could end the destructive effects of SMA once and for all. It is possible that over time the price will drop and the drug will become more affordable.
This not only stops the development of the disease, but also improves the patient’s health. Only one dose of Zolgensma (Onasemnogene abeparvovec) is enough to cure people suffering from this ailment, but there is an important requirement for the treatment to be effective: the age of the patient must not exceed two years, that is, it is used only in babies.
The injection process takes an hour, and then the medicine does the rest of the work on its own.
The title of the most expensive drug in the world is not empty words: Zolgensma was even listed in the Guinness Book of Records. Due to the difficulties associated with its enormous price, in some cases, benefactors donate a considerable part of the sum or even the entire amount to families so that they can give a new life to the sick.
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In December 2019, the company that produces Zolgensma decided that it would donate 100 doses of the drug in 2020 to randomly selected patients. In order to participate, the treating physician must send a request to the production company attaching documents that confirm the diagnosis.