One such condition is Spinal Muscular Atrophy (SMA), a genetic disorder affecting infants and young children, impacting their movement and muscle function. SMA is a genetic disorder of movement ...
The mutation that causes Spinal Muscular Atrophy (SMA) can be detected in a prenatal screen. But a parent's decision to test for this terrible disease is not as easy as you would think.
Affan, seven, and Erhan, five, have Spinal Muscular Atrophy (SMA ... for genetic conditions like SMA and Duchenne Muscular Dystrophy (DMD) - which causes muscle weakness and degeneration.
such as the development of transformative drugs for spinal muscular atrophy, and the starting of UK-first trials of gene therapy for Duchenne muscular dystrophy. I’m excited to see what further ...