Caracas Chronicles

Photos: Juan Diego Vílchez Valbuena

Marlyn Roldán Valencia lives in Quíbor, 20 minutes from Barquisimeto and four hours from Caracas, with her 10-year-old girl diagnosed with Hurler Syndrome. She’s one of the many mothers who fights for this specific medicine to arrive to Venezuela. Children with Hurler Syndrome have problems with their extremities, deformities in their skeleton, cognitive decline, characteristic facial traits, dwarfism, heart diseases and a reduced life expectancy. If they don’t get a timely treatment, they die of respiratory problems caused by the accumulation of mucus in their lungs.

Only the State can import the medicine that patients with Hurler Syndrome require. Patients usually receive assistance through the International Charitable Access Program (ICAP), “A program regulated by Sanofi Genzyme’s global reference framework, in which the population eligible to access these benefits are pediatric patients under five years old diagnosed with an MPS type I pathology, in the Hurler variant. Each case is analyzed by an independent and international multidisciplinary medical board which ultimately decides on incorporating the patient to the program.” However, this program isn’t enough to cover the demands of those affected.

In the country, there are 137 people with Guacher, Fabry and Pompe; rare diseases that are also treated with high-cost therapies. There is a total of 14 diagnosed cases of Hurler.

Only the State can import the medicine that patients with Hurler Syndrome require.

So far in 2018, two children have died in Lara’s Venezuelan Institute of Social Security (IVSS), which provides treatment for eight patients with Hurler Syndrome in Portuguesa and Barinas. Out of these eight cases, a girl died due to alleged medical malpraxis in Barinas, while the other five await for the medicine.

Marlyn often cries, knowing that her girl’s disease may get worse at any moment. Originally, her daughter “suffered changes in her body: she lost hair on her skin and her head,” she said.

Gregoria Sameco, director at the Venezuelan Association of Patients with Lysosomal Diseases (Avepel), herself a Gaucher patient, is concerned by the fact that the State still hasn’t acquired the treatment for Hurler patients. During the interview, she noted that as a non-government organization, they won a lawsuit against the IVSS to bring the medicine to Venezuela. “Back then we succeeded: we received the benefit freely and obligatorily. But only a few started receiving the treatment since 2009, and since 2016 no medicines have been received.” She said that “we have the  legal right to healthcare, and if we don’t get the treatment our quality of life will decline more and more until we die. As Venezuelans and patients of these pathologies, we call for the IVSS and the government to give us our treatment.”

Marilyn explains the importance of the treatment for Hurler. During the treatment, her daughter had better mobility in her joints and even “lived normally for six years. But in 2016, the treatment started failing intermittently until it disappeared.” With the medication, Marilyn’s girl could attend school, but that’s no longer possible, because she lacks the physical conditions to do that.

During the treatment, her daughter had better mobility in her joints and even “lived normally for six years.

In a visit to IVSS headquarters in Caracas, they told her that other diseases were a priority, but even so, she’s still fighting. “They told us that the purchase was impossible, because there was a larger group of cancer patients who needed treatment. Their demands were stronger than ours. Back then, there were only four children, but then others joined until they reached eight patients in the Central-Western region and we’re fighting to prevent losing the treatment,” she said.

Marilyn must do the impossible to find the medicine: from requesting it on social networks to trying to talk with politicians. When she was in the IVSS in Caracas, “they told us to wait for current Lara governor Carmen Meléndez to consolidate the project. We even got in touch with them through Avepel, and they told us to participate in events while she was in electoral campaign.” But in the end, nobody answered their plights.

She’s aware of the complex humanitarian emergency in Venezuela, but her daughter isn’t. “She asks me every day when she’s going to resume her treatment, because she knows and is aware that the medicine helps her feel better. What explanation can I give her? How can I make her understand that it’s not something that I or the doctors don’t want to give her, but that the country’s situation makes it hard to acquire?” she said in tears.

Other protagonists, same story

Amanda Ruiz gives her five-year-old daughter with Hurler the best possible care in Los Cerritos sector in Tintorero, half an hour from Barquisimeto.

She fears that her daughter could die at any moment, because she was recently hospitalized due to respiratory problems. The treatment, which only the State can buy, is out of her reach too.

She fears that her daughter could die at any moment, because she was recently hospitalized due to respiratory problems.

Since her daughter stopped receiving the medication, she gets sick often. “The doctor extracts lots of phlegm from her lungs,” Amanda says, adding that her girl’s organs

A 10-year-old girl died in La Batalla area, western Barquisimeto, after months of not getting her medication. Yaquelín Gómez is now a spokeswoman for all the mothers who struggle so that their children have a better quality of life.

Yaquelín went to the pharmacy at the Dr. Pastor Oropeza hospital, like other families, to receive false promises: “For a month they told me to go during the week, they kept telling me that until my girl died,” she said.

Her daughter was referred to the IVSS by Dr. Pedro Estrada, from the UCLA’s Medical Genetic Unit, on June 2, 2009, ten months after she was born. She frequently suffered respiratory impairment, which eventually killed her in the Dr. Agustín Zubillada University Pediatric Hospital (HUPAZ), where she remained hospitalized for four days before dying from pneumonia.

The second death caused by lack of Hurler treatment, took place in Quíbor, El Calvario, a sector where people live in ramshackle clay houses.

María Gil and Jeison Daza said farewell to their eight-year-old son. He died of respiratory problems. “When he was a year and a half, he started getting the dose, drop by drop,” they said.

He was diagnosed when he was three years old, thanks to her grandmother’s curiosity, who saw another children with characteristics similar to those of her relative while riding a bus. They took him to the pediatrician to be diagnosed with Hurler. “We didn’t know that disease. Nobody told us anything,” they said.

Since January 2017, there’s no Hurler treatment in the Dr. Pastor Oropeza Hospital in Barquisimeto.

Since January 2017, there’s no Hurler treatment in the Dr. Pastor Oropeza Hospital in Barquisimeto. Sanofi Genzyme’s Communications manager in Venezuela, Narvin Delgado, refused to provide figures of the files of patients who received the medicine according to reports from the IVSS, as well as the investment made by the Venezuelan State. She merely said: “In this case I think you must talk directly with the appropriate authorities and request this information.”