"Two babies have received the first-ever gene therapy for Tay-Sachs disease after over 14 years of development."
Tay-Sachs disease, a rare genetic disease passed from parent to child, poses a problem when infected individuals lack enzymes that are needed to break down fatty substances. Signs and symptoms occur from 3-6 months of life. Symptoms then progress and further lead to death, around age 4 or 5.
"In 1887, American neurologist Bernard Sachs described the profound neurological symptoms of Tay-Sachs in a seminal paper:
“… Nothing abnormal was noticed until the age of two to three months, when the parents observed that the child was much more listless than children of that age. … The child would ordinarily lay upon its back, and was never able to change its position … it never attempted any voluntary movement … the child grew steadily weaker, it ceased to take its food properly, its bronchial troubles increased, and finally, pneumonia set in, it died August, 1886.”
The terrible symptoms of Tay-Sachs disease are truly hard to read about... but it is relieving to know that this historic advancement in healthcare, specifically the healthcare of children, has been made. What are your thoughts? What similar advancements do you think are on the horizon?
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