Daughter with Biotinidase Deficiency

The story of Sarah

On November 3rd, 2019 our daughter, Ava Rose Leone, was born. She is our third and our last child. She is the completion to our family of five. On the day Ava came into the world she had a heel prick in the hospital which we really had no idea what that little heel prick checked for and I don’t think many parents do. That heel prick tests for a long list of diseases and luckily NY state was one of the states that allows testing for Biotinidase deficiency.

        We found out on November 7th from a phone call from our pediatrician after we were already home from the hospital that Ava had been diagnosed with this rare disease.  In that moment, time stood still. My husband and I had our pediatrician on speaker as we listened to him explain the disease to both of us and how only 1/60,000 have it. We were silent taking it all in. One second everything seemed completely normal and three healthy kids to learning something was missing in Ava. She lacked an enzyme, (later we found out she only lacked it partially).  The second we hung up the phone, my husband and I held each other and cried. We hated knowing our perfect little baby had something missing. We started doing research inmediatly and had to meet with a specialist later that week. We quickly learned that Ava will be completely fine and hopefully avoid all symptoms as long as she took Meribin (a pure form of Biotin) everyday for the rest of her life.

     My husband and I knew we were lucky there was a way to avoid the long list of symptoms of BD, which include seizures, hair loss, skin rashes, Atonia, muscle weakness, cognitive delays, developmental delays, etc. However, there are states that don’t test for this in the heel prick at the hospital. I think some states think it’s not worth the tax dollars to have testing done for a disease that is so rare. That scares me. It makes me think of all the families with children with BD who have suffered because they didn’t catch it at birth. Every day that a child with BD is not taking biotin, it’s effecting them.  I have already been in contact with some families through social media that have children that have suffered seizures and are severely delayed because they weren’t diagnosed at birth. 

     It is so important to raise awareness of these rare diseases. We need more research and more answers. We need more testing to be done worldwide.  We need to help avoid these diseases from happening when we know there is a test out there to diagnose it early on.  I hope my story reaches a few people at least.  Maybe another parent will look this up and think of the day they found out their child had biotinidase deficiency.  Please reach out to me if you’d like to at [email protected]

     Ava is 3 months old now and so far she is living a healthy and normal life. She is smiling, cooing, and able to hold her head up strong and has great muscle control of her neck and head. We have been giving her the meribin since she was a week and a half old. We are hopeful for the future.