About Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder caused by mutations in the SMN1 gene, leading to progressive loss of motor neurons in the spinal cord. It is one of the most common fatal genetic diseases of infancy. Types range from severe infantile-onset (Type 1) to adult-onset (Type 4). Disease-modifying treatments now exist: nusinersen (Spinraza), onasemnogene abeparvovec (Zolgensma), and risdiplam (Evrysdi). Clinical trials remain highly active, especially in gene therapy and next-generation SMN-targeting approaches.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Spinal Muscular Atrophy trials.
The SMN1 gene test is the definitive diagnostic — ask your neurologist for genetic confirmation before enrolling in any trial.
SMA trials often stratify by SMN2 copy number — know your copy count as it affects eligibility for many studies.
Newborn screening for SMA is now available in many countries — pre-symptomatic treatment significantly improves outcomes.
Patient Resources
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