spinal muscular atrophy on blue

spinal muscular atrophy sma genetics neuromuscular disease disorder inheritance mutation smn1 smn2 gene protein motor neurons muscle weakness degeneration symptoms diagnosis prognosis treatment therapy spinraza zolgensma risdiplam fda approval clinical trials research cure hope management support family caregivers infants children adults type severity onset life expectancy breathing mobility equipment physical occupational nutrition speech assistive devices wheelchair genetic counseling carrier testing awareness advocacy community organizations funding empowerment