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From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of <i>SOD1</i> Mutated Amyotrophic Lateral Sclerosis

Willeke van Roon‐Mom, Chantal Ferguson, Annemieke Aartsma‐Rus

2023Nucleic Acid Therapeutics52 citationsDOIOpen Access PDF

Topics & Concepts

Food and drug administrationMedicinePharmacologyNeurogenetic and Muscular Disorders ResearchAmyotrophic Lateral Sclerosis ResearchCancer-related gene regulation
From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of <i>SOD1</i> Mutated Amyotrophic Lateral Sclerosis | Litcius