In June, the U.S. Food and Drug Administration approved the medication fenfluramine for people with severe epilepsy and Dravet syndrome, or SCN1A-related seizure disorders, who are 2 years of age or ...
"I dropped onto the sidewalk, my arms wrapped around my bent knees. 'Take him inside, don’t let him see me,' I blurted out.” ...
Despite treatment with standard-of-care anti-seizure medicines, children with Dravet syndrome experienced high seizure burden and plateaued in ...
BEDFORD, Mass.--(BUSINESS WIRE)--Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression ...
(BPT) – Grace is a gift. At eight years old, her infectious exuberance for life flashes in her big brown eyes and cheeky smile. She’s also a warrior. You would never know that at just six months old ...
Two first-time parents have felt both heartache and hope in raising their medically complex son. M and her husband D, who prefer not to use their full names, had worked hard to start a family. When ...
(BPT) – Grace is a gift. At eight years old, her infectious exuberance for life flashes in her big brown eyes and cheeky smile. She’s also a warrior. You would never know that at just six months old ...
BEDFORD, Mass.--(BUSINESS WIRE)--Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression ...
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