What is the NSAA and when is it applicable?

The North Star Ambulatory Assessment has 17 items scored 0–2, ranging from standing and walking to jumping and running. It is used for ambulatory DMD patients typically aged 4–16 years. Loss of ambulation correlates with a drop to 0 on most items.

How do exon-skipping therapies affect DMD functional scores?

Eteplirsen (exon 51 skipping) and similar drugs aim to restore partial dystrophin expression, potentially slowing functional decline. Functional scores are primary endpoints in ongoing clinical trials for newer exon-skipping therapies.

Duchenne MD Functional Score

Assess functional ability in Duchenne Muscular Dystrophy using the North Star Ambulatory Assessment (NSAA) and upper limb functional scales.

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Frequently Asked Questions

What is the NSAA and when is it applicable?

How do exon-skipping therapies affect DMD functional scores?