"With tears in our eyes and great joy in our hearts, we'll let you know that Spinraza has just been approved by ANMAT! For AME type 1, 2 and 3a without limitation. We can not over emotion!"Saying the emotional message that patients' relationships have with them back muscle atrophy (AME) and is expected to give a long time ago.
Comfort Following year on year there is a claim for families of people with SMA, a national genetic disease that affects children but also adults and counts in Argentina about 300 cases. They made a number of parade in the last one that took them from the Health portfolio to the Casa Rosada to ask that the ANMAT End patient registration to access treatment.
Characterized by the loss of aggressive and advanced muscle strength, this pathology causes the weakness and atrophy of the voluntary muscle that is responsible for basic functions such as cropian, walking, controlling the throat, eating and something as simple and essential as breathing.
"It's a disease that kills, In 55% of cases, children die before they reach two years", warned by the Argentine Family Association AME and said that Spinraza (had expanded by the international laboratory Biogen) "This is the first treatment and only". "Patients often say they feel they are transforming themselves into living statues, the disease is so cruel," explained the association.
What the drug does
Y nusinersen (known as Spinraza better) is an innovative medicine to treat SMA. It was in the research period until December 2016, when it was approved by the United States and then by the European Union after it proves that it prevents the disease from giving it up, although it is still not known if the effect will be held over time.
The drug he demonstrated it effectively in patients with SMA type 1 (one of the most aggressive) and also show progress control in type 2 and SMA type 3 in each case: baby, boys, teenagers and adults. It's one of the most expensive drugs that exist, with a value of nearly 150 thousand dollars per dose.
But the relatives' battle has one other step. "Still we are waiting for the Scoping Scheme that the Ministry of Health will have. This is the result of great effort for everyone, there is still a long way to go, but the approval in our country gives us more strength to continue fighting. Until #SpinrazaPara Everything we will not stop! "They celebrate.