The days of waiting and anguish are over for Agustin Cordenos Garces and his family, since this Saturday a four-year-old child received his medicine imported from the United States for treatment. achondroplegiaIts cost is about 30 thousand dollars per month.
Achondroplasia is the most common form of dwarfism. This means that the person has a chromosomal bone disorderThat is, all long bones are shortened symmetrically. In these cases, the length of the spine is normal, which causes the body to grow disproportionately.
“We are very happy. The wait was getting too long because almost two months have passed since the verdict,” he says. bugle The child’s father, Pablo Andres Cordenos (36).
And he hopes to clarify: “From Monday they will be doing Agus injections in the city of Cordoba, we will be there for a week and then we will start doing them at home.
Augustine lives in Canals, Cordoba. The medicine you will start using every day is called vosorticide And its trade name is Voxzogo. It helps to stimulate the growth of diseased bones.
Agustín Cordenos Garces is three years old and suffers from achondroplasia. Justice has authorized a drug to treat him.
Agustin will have to inject until growth plateswhich usually happens in men between the ages of 17 and 18. They are imported under the exceptional access regime for unregistered medicines provided by ANMAT.
“we are fighting ANMAT has approved this drug in our countrySo other children can access it without going through the legal process,” he says. And he says there are already 30 families asking for the drug, and they have to go through a legal process to get it.
Faced with the impossibility of not being able to pay for his care, last December, Pablo began a lawsuit in court. The trial court ruled that the cost of the injection would be borne by the Department of Health and the Superintendent of Health 50% and half social work and prepaid.
But the decision was not final and the Federal Chamber of Cordoba decided that the Ministry of Health takes charge of the purchase of the drug and then coordinates social work and part of the reimbursement by prepayment.
” a few days ago final sentence Where justice requires that 90% of the cost of the appeal be paid by the State, 5% by social work and the remaining 5% prepaid”, explains Pablo.
Last year, the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) Approved the use of this drug Treat children with pathology.
“In the European Union it is approved to be given from the age of two and in the United States from the age of five. And there are studies now looking to demonstrate its effectiveness from the first months of life” , said Cordenos.Told this newspaper two months ago
On this occasion, the boy’s father clarified that I don’t know the long term effects Because the approval of the drug is recent. And he explained: “It is likely that this contributes to the prevention of associated malformations such as cervical stenosis (vertebral constriction), for which the agus had to be operated on once every 10 months.
There was another case in Mendoza of a six-year-old girl who had also received medication for achondroplasia. Civil Judge Marcela Ruiz Diaz ruled in favor of the family and ordered their prepaid pharmaceutical company to cover 100% of the costs.
The importation of the remedy must be immediate because the magistrate took into account the fact that achondroplasia causes a disability that worsens over the years and affects quality of life.
Achondroplasia in both children is caused by modification of genetic information It receives the growth factor receptor from fibroblasts, the cells that grow bone lengthwise.
the one who produces pathology in cartilage development, with accelerated calcification that inhibits normal bone growth. People with this deformity have a normal sized torso, short limbs and a slightly larger head.
Appears like spontaneous mutation, which happens every 20,000 births. About 90% of children with achondroplasia do not have parents with pathology. In turn, achondroplasia usually causes recurrent ear infections, spinal problems, leg sprains and apneas.
Life expectancy and IQ of people with pathology they look like normal sized peopleHowever, children with this problem often have slow motor development as infants due to their body proportions.