A Therapy for Most with Cystic Fibrosis
1Program of Molecular Medicine and the Program for Individualized Therapy at SickKids Hospital (CFIT), The Hospital for Sick Children, Toronto, ON, Canada; Departments of Physiology and Biochemistry, University of Toronto, Toronto, ON, Canada.
Abstract:
TRIKAFTA is the third drug approved by the FDA that rescues defects caused by the major mutation F508del. It is superior to its predecessors that were approved for patients who are homozygous for F508del because TRIKAFTA is also effective in CF patients who harbor only one copy of this mutation.
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