FIRST APPROVED TREATMENT FOR EPIDERMOLYSIS BULLOSA
ETHAN, 7 YEARS OLD, IS THE ONLY CHILD IN ARUBA WITH THIS CONDITION
At the end of the week, we received the news that the Food and Drug Administration (FDA) in the United States has approved a topical gene therapy as a first treatment for wound healing in patients with Dystrophic Epidermolysis Bullosa (DEB). The approved medication is called Vyjuvek, which is a type of genetic therapy that directly targets the underlying cause of this condition.
How is Vyjuvek applied?
Vyjuvek comes in the form of a gel/ointment that is applied by a healthcare professional to the wounds of DEB patients once a week, or the frequency of application depends on the patient's age.
The approval of this came after a long period of more than 4 years of trial testing for its effectiveness. The compiled data on real results were presented to the FDA, which evaluates them before granting approval. With the approval of Vyjuvek, it means that the remedy will soon be distributed and made available to all patients who can obtain a prescription from their doctors, and it will also become applicable for coverage by medical insurance in the United States.
Dystrophic epidermolysis bullosa (DEB) is a condition resulting from a genetic defect that affects the texture of the skin, making it extremely fragile and prone to blistering even with minor trauma. The severity of this condition varies from patient to patient, ranging from mild cases with blisters in specific areas to more severe cases where the blisters can occur throughout the body, including internally. This has led to these children being referred to as "butterfly children."
Although cases of Dystrophic epidermolysis bullosa (DEB) are rare (1 case in 1 million children), Aruba also has one case of a boy named Ethan, who is 7 years old and was born with this condition.
WHAT DOES THIS MEAN FOR ARUBA'S BUTTERFLY BOY, ETHAN?
Aruba falls under European regulations, and this approval in the United States does not necessarily mean immediate availability in Aruba. However, considering Ethan's case is a "rare" condition, there may still be a way to navigate and obtain access to the treatment.
Among the five types of EB that exist, Ethan has Recessive Dystrophic Epidermolysis Bullosa, which can range from severe to milder forms. Ethan falls into a more concentrated category where he has certain areas that are consistently affected by blisters.
Ethan has his own Facebook page called "Cares for Ethan" where regular updates are posted about his condition. The most recent update is about the GTUBe that has been placed to assist with his feeding process. Although Ethan has a relatively liberal diet and can eat what he desires, often due to blisters in his mouth and throat, eating becomes challenging for him. With this tube, Ethan receives the necessary assistance to ensure he gets proper nutrition during these difficult moments, even when he is sleeping.
We spoke with Shahaira Bolongaita Geerman, a nurse herself and Ethan's mother, about what this means for her son. She has already been in contact with the DEBRA organization, which is dedicated to the EB community and has informed her that they are awaiting information about the distribution in order to provide more details on how to obtain this remedy. "I have also been in touch with parents of children in the Netherlands who are waiting. We are all excited because this is finally good news for our children with this condition."
Often, the survival of "Butterfly" children is not long, estimated at 30 years, although there are some cases that have surpassed this age. So far, there is no cure for EB. Although many of these children grow up and try to live as normal a life as possible, the severity of this condition can cause a lot of pain in their daily lives.
With the approval of this treatment, which can help heal wounds more quickly, it also means that the chances of infection may decrease. Hopefully, this will have a positive impact on their survival in the long run.
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