06Jul 2023
Cystic Fibrosis: From Tragedy to Triumph

Cystic Fibrosis: From Tragedy to Triumph

New breakthroughs in the treatment of cystic fibrosis (CF) offer hope for young children. Highly effective modulator therapies (HEMTs) have been developed to correct gene variants causing CFTR protein defects. Studies demonstrate the safety and efficacy of HEMTs in children as young as 2 years old, paving the way for early intervention after diagnosis through newborn screening. Long-term follow-up data also show sustained benefits of HEMTs in older children. However, rare side effects like benign intracranial hypertension (BIH) have emerged, emphasizing the need for postmarketing surveillance. Further research aims to address nonresponsive mutations and expand access to HEMTs worldwide, particularly in underserved communities.

  • #critical care

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