MSP changed its mind and bought medicine for Luciano Supervielle's daughter

2023-09-16 03:08:11

First, it seemed like a victory for children’s rights. Nina, daughter of Luciano Superviellewhom Justice favored by ordering the State to provide him with the treatment Voxzogo, but then the Health authorities appealed the ruling. A bucket of cold water for her family.

Every day that Nina is without treatment, says Luciano, is a day lost in her chances of having a better quality of life that the Voxzogo treatment can provide. This expensive drug, which has a price of US$10.000 por dosisis a C-type natriuretic peptide (CNP) analog approved to increase linear growth in individuals or children with achondroplasia aged 5 years and older with open epiphyses (growth plates).

It must be administered once a day, so a month-long treatment is priced at US$300.000out of reach of practically any family.

The authorities of the Ministry of Public Health (MSP) confirmed this Friday that, despite the ruling issued by Justice, the entity has already acquired 30 vials of the Voxzogo medication intended for the 9-year-old daughter of musician Luciano Supervielle, Nina.

During a press conference, the undersecretary of the MSP, José Luis Satdjian, emphasized that they are waiting for the judicial decision, since this instance will determine whether or not treatment is granted to the patient. He added that the ministry is immersed in a system of laws and regulations “which are what we have, which we will like more or less and that perhaps it is a good time to improve it and we need some legal impetus to correct some issues.”

The official highlighted that there are precedents of patients who, after a protection process, have initiated ordinary lawsuits to obtain access to medication. Finally, the undersecretary emphasized that the medicine was prescribed by a doctor from the Social Security Bank (BPS) and mentioned the inclusion of provisions in the Accountability to transform the National Reference Center for Rare Diseases (Crenadecer) into a place where similar cases are resolved.

“I just found out that @MSPUruguay bought the first doses for my daughter’s treatment. It is deeply exciting and important for me, for Nina and for the children who come after. My gratitude is infinite,” she wrote on the social network X Supervielle. “The expressions of affection and empathy that I have received these days are a gift that will accompany me forever. Thank you @juanceretta for guiding me through this process,” she added.

What is Voxzogo and how does it help in achondroplasia?

Voxzogo is the trade name of the drug vosoritide, the first and only one approved in the world to treat people with achondroplasia. This inherited genetic disorder affects bone growth and causes dwarfism. It is characterized by short limbs, significantly shorter than average stature, and long bone deformities.

This disorder is due to a mutation in the FGFR3 gene, which regulates bone growth. People with achondroplasia usually have an average-sized head, similar to that of people without the disorder, and a life expectancy that matches that of the average of the population in which they reside. However, they may face medical challenges, such as spinal problems or breathing difficulties. Treatment often includes physical therapy and, in some cases, surgery to address orthopedic complications.

Voxzogo (vosoritide) is a daily injection that has been approved as an analog of C-type natriuretic peptide (CNP). Its goal is to promote linear growth in individuals, especially children, ages 5 and older, who suffer from achondroplasia and still have open epiphyses, that is, active growth plates.

Children being treated with Voxzogo should undergo regular evaluations to monitor their weight, physical development, and growth rate.. The dose of the medication is adjusted accordingly as treatment progresses. Importantly, Voxzogo should be discontinued once it is confirmed that there is no further growth potential and the growth plates have closed.

It is important to note that the use of Voxzogo can lead to serious side effects in some patients, including a temporary decrease in blood pressure. To reduce the risk of experience a drop in blood pressure and related symptoms such as dizziness, fatigue or nauseait is essential that patients are well fed and hydrated approximately one hour before receiving the Voxzogo injection.

Those who wish to learn more about the treatment, should be referred directly to a specialized medical professional and carefully read the manufacturer’s brochures, available here in English.