La única cura para la dolorosa enfermedad de células falciformes hoy en día es un trasplante de médula ósea.
Pero pronto puede haber una nueva cura que ataque el trastorno en su origen genético. El martes, los asesores de la Administración de Alimentos y Medicamentos revisarán una terapia genética para la anemia drepanocítica, que es hereditaria y en Estados Unidos afecta principalmente a los negros. Las cuestiones que considerarán incluyen si se necesita mayor investigación sobre las posibles consecuencias indeseadas del tratamiento.
Algunas personas dirán que es como ser apuñalado por todas partes”. El trastorno afecta la hemoglobina, la proteína de los glóbulos rojos que transporta oxígeno. Una mutación genética hace que las células adquieran forma de media luna, lo que puede bloquear el flujo sanguíneo y causar un dolor insoportable, daño a órganos, derrames cerebrales y otros problemas. Millones de personas en todo el mundo, incluidas unas 100.
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