For the final 10% of cystic fibrosis patients, the breakthrough still hasn’t come, the article reports.
Modern drugs have revolutionized treatment for the vast majority of people with the condition.
But those advances bypass patients with specific, rare genetic mutations.
Genetic profiles dictate treatment success, and rare mutations fail to respond to blockbuster drugs.
Rare mutations often lack commercial backing, and clinical trials for these groups remain limited.
Supportive care remains the main strategy for managing severe symptoms in these patients.
Researchers are exploring alternative gene-editing techniques to close the gap.
Advocacy groups are urging more funding for these overlooked patients.
Read the full article for more details on the remaining cystic fibrosis patients.
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