DDNews features Taconic Biosciences' Dr. Megan MacBride who gives remarks on in-vitro models and their limitations:
"The 10-year-old boy stares up at the ceiling from his bed, a crooked smile crossing his face.
His parents come and go, checking on him periodically to make sure he is OK. They know they won't disturb him; he doesn't really see them. And even when he does, there is little behind those eyes to suggest recognition.
The boy has Hurler Syndrome, the most severe form of mucopolysaccharidosis type I (MPS I). It is a condition that has effectively left him in a vegetative state, an infant in a preteen body--a life challenged by the lack of a single enzyme.
Enzyme replacement therapy (ERT) helps with some of the symptoms. It's likely that it has allowed him to live this long."
Read the complete article at: DDN-News.com
His parents come and go, checking on him periodically to make sure he is OK. They know they won't disturb him; he doesn't really see them. And even when he does, there is little behind those eyes to suggest recognition.
The boy has Hurler Syndrome, the most severe form of mucopolysaccharidosis type I (MPS I). It is a condition that has effectively left him in a vegetative state, an infant in a preteen body--a life challenged by the lack of a single enzyme.
Enzyme replacement therapy (ERT) helps with some of the symptoms. It's likely that it has allowed him to live this long."