Brain drugs can now cross the once impenetrable blood-brain barrier
Briefly

Daiza Gordon experienced immense loss when her brothers died from Hunter syndrome, a genetic disorder affecting boys. At just 19, she felt guilt after failing to save her youngest brother. Now, she faces a similar plight as her three sons have also been diagnosed. However, her children are part of a clinical trial testing a groundbreaking treatment that delivers the missing enzyme into the brain. Early results suggest significant cognitive and physical improvements, fueling her hope that her sons can achieve milestones previously deemed impossible.
Gordon's two sons, who are participants in a clinical trial aiming to deliver a replacement for the missing enzyme, are showing remarkable improvements in their development, defying the previous grim prognosis for Hunter syndrome.
Gordon reflects on her past with her brothers' deaths due to Hunter syndrome and expresses hope regarding her sons' futures, now that they are responding positively to a new treatment that allows enzyme delivery into the brain.
Read at Nature
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