The most expensive drug in the world. Zolgensma could save Alex’s life

There has never been such a huge collection in the history of the Siepomaga portal. Little Alex’s parents collect PLN 9 million for the most expensive medicine in the world. Zolgensma is an innovative drug approved for the treatment of spinal muscular atrophy. Four-month-old Alex has a chance of recovery, but the amount his parents have to raise is knocking out.

Zolgensma – the most expensive drug in the world

Zolgesma is a drug that was approved by the American Food and Drug Administration (FDA) in May 2019 for use in children under two years of age suffering from spinal muscular atrophy (SMA). The drug is produced by the Swiss medical concern Novartis. The cost of one-time Zolgesma therapy is approximately $ 2,1 million, or almost PLN 9 million.

Such a high price of the drug was initially the reason for the FDA’s reluctance to market the drug, but the manufacturer argued that a single infusion could save patients’ lives and thus save money that would have to be spent on long-term treatment.

Therapy involves replacing the damaged SMN1 gene with a working copy. In the disease of spinal muscular atrophy, the patient has the SMN1 gene missing or mutated. For this reason, patients do not produce enough of the motor neuron survival protein, which plays a key role in their maintenance. Patients with type 1 type XNUMX SMA require the most intensive and symptomatic treatment. They are unable to sit unsupported and usually do not survive beyond two years without respiratory support.

One dose of the drug is enough to stop the development of spinal muscular atrophy. As reported by Businessinsider, a patient can pay $ 2,1 million for a drug at a time or spread the payment into 5 installments of $ 425 each. dollars.

The most expensive drug in the world could save Alex’s life

Zolgesma was approved in the United States on May 24, 2019. The approval of this drug is still ongoing in the European Union and Japan. It will probably happen at the end of 2019.

It is this world’s most expensive drug that could save Alex’s life. The largest collection in its history is taking place on the Siepomaga website. PLN 9 million is needed for treatment! The boy already has almost PLN 3 million on his account, but it is still not enough to start treatment.

The boy was born at the end of June. His mother recalls that pregnancy and childbirth were almost textbook-like. At birth, Alex scored 10 points on the Apgar scale, and there was no indication that he was terminally ill. After about a month, his condition began to deteriorate. He stopped moving his arms and legs, “overflowing” through his hands. Alex was hospitalized and genetic testing revealed a terrible diagnosis.

Alex suffers from Spinal Muscular Atrophy – Type 1 SMA. This is the worst form of the disease. The very expensive therapy in the United States is a hope for the child. A drug that has been authorized a few days before a boy is born could save his life.

Currently, Alex is undergoing rehabilitation, he is also taking another drug, Spiranza, reimbursed in Polandwhich slows the progression of the disease. But it won’t get Alex cured. Anyone who wants to contribute to the collection for Alex’s treatment can donate money to the Siepomaga website.

You can read about Alex and his family here: Record collection for Siepomaga. Little Alex needs PLN 9 million. This story cannot be ignored

See also:

1. The five most expensive drugs in the world!
2. Americans cure cancer. Poles are dying of cancer
3. The most promising heart regulating devices

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