NACFC 2025 | W17: Extrapulmonary Manifestations & Mechanisms
Автор: Cystic Fibrosis Foundation
Загружено: 2025-12-03
Просмотров: 22
High-efficiency CFTR modulator therapies (HEMTs) have revolutionized the treatment landscape for people with cystic fibrosis by targeting the underlying CFTR protein defect. These therapies have led to significant improvements in pulmonary function, quality of life, and life expectancy. However, their effects on extrapulmonary manifestations of CF are more variable and remain incompletely understood.
As individuals with CF live longer, new extrapulmonary complications may emerge, reflecting both the effects of chronic disease and aging, as well as unanticipated consequences of long-term HEMT use. While some extrapulmonary symptoms may improve, others may persist or even develop de novo.
Despite the substantial benefits of HEMTs, their long-term impact on specific organ systems—including the brain, liver, pancreas, kidneys, and reproductive tract—remains under active investigation. This underscores the critical need for ongoing research, longitudinal clinical monitoring, and mechanistic studies to fully understand the evolving phenotype of CF in the era of modulator therapy.
Originally recorded Oct. 24, 2025. Recording may have been edited for time and/or content.
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