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3 diseases ยท Inherited genetic conditions and chromosomal disorders
Fatal genetic disorder causing progressive destruction of nerve cells in the brain and spinal cord. No treatment or cure.
Inherited blood disorder causing misshapen red blood cells, pain crises, organ damage, and shortened lifespan. Disproportionately affects people of African descent.
Fatal genetic muscle disorder affecting boys. Progressive weakness leads to loss of walking, breathing, and death typically by age 30.