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Pharmaceutical
Rare and Orphan Diseases
SMA, Duchenne and Neuromuscular Diseases Pipeline
Code: NSC/PHRM/0012Published Mar 15, 2026Data window: 3 months
About this report
Spinraza, Evrysdi, Zolgensma, Elevidys, Sarepta, Roche: second wave of gene therapies.
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