It is a genetic disorder characterized by weakness and wasting (atrophy) in muscles used for movement (skeletal muscles). The severity of symptoms and age of onset varies by type. Some types are apparent at or before birth while others are not apparent until adulthood.
Nerve cells in the brain stem and spinal cord that control essential skeletal muscle activity such as speaking, walking, breathing, and swallowing, leading to muscle weakness and atrophy.
Inaugural fundraising event for Spinal Muscular Atrophy research
Read MoreWe aim to develop novel therapies based on individual’s own specific genotype, a process to identify diseased causing genes and then to develop individualized treatments for Spinal Muscular Atrophy (SMA), also known as floppy baby syndrome, which is a neuromuscular disease.
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