What functional scales are used for different SMA types?

CHOP-INTEND is used for SMA Type 1 (non-sitters). The Hammersmith Functional Motor Scale Expanded (HFMSE) is used for Types 2–3. The Revised Upper Limb Module (RULM) captures upper limb function in non-ambulant patients.

How do nusinersen, risdiplam, and onasemnogene abeparvovec affect SMA functional scores?

All three disease-modifying therapies have demonstrated meaningful improvements in functional motor scores in clinical trials, with the greatest gains seen in presymptomatic or early-treated patients. Baseline functional scores inform treatment urgency.

SMA Functional Outcome Measure

Assess functional motor abilities in Spinal Muscular Atrophy using the Hammersmith Functional Motor Scale or CHOP-INTEND for infants and young children.

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Frequently Asked Questions

What functional scales are used for different SMA types?

How do nusinersen, risdiplam, and onasemnogene abeparvovec affect SMA functional scores?