Long-Term Prognosis of Krabbe Therapies: Shifting Expectations for Patient Survival and Quality of Life
The long-term prognosis of Krabbe disease has historically been grim, with infantile-onset cases typically leading to death by age 2-4. However, emerging therapies—including improved HSCT protocols, enzyme replacements, and gene therapies—are transforming expectations, offering hope for extended survival and improved quality of life. Understanding these shifts is vital for patients,...
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