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SPINRAZA (nusinersen)

Spinal Muscular Atrophy

ApprovedPartnered/marketed

Key Facts

Indication

Spinal Muscular Atrophy

Phase

Approved

Status

Partnered/marketed

Company

Ionis Pharmaceuticals

About Ionis Pharmaceuticals

Founded in 1989 by Stanley T. Crooke, Ionis Pharmaceuticals is a pioneer in RNA therapeutics with over three decades of innovation in antisense oligonucleotide technology. The company has successfully transitioned to a fully integrated biotechnology organization, launching its first independent medicine TRYNGOLZA (olezarsen) for familial chylomicronemia syndrome. With six currently marketed medicines discovered and developed by Ionis, the company maintains a rich late-stage pipeline focused primarily on neurology and cardiology, while continuing to advance breakthrough RNA-targeted therapies that have redefined treatment for serious genetic diseases.

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Therapeutic Areas

Neurology Cardiology Rare Diseases Metabolic Disorders Genetic Diseases

Other Spinal Muscular Atrophy Drugs

Drug	Company	Phase	Status
GYM329	Chugai Pharmaceutical	Phase 2	Active
Spinraza	Royalty Pharma	Commercial	Active
Evrysdi	Royalty Pharma	Commercial	Active
Spinal Muscular Atrophy Program	Biogen	Commercial	Active
ASO Candidates for SMA	Biogen	Pre-clinical	Active