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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 SOD1 Mutated Amyotrophic Lateral Sclerosis.

Willeke M C van Roon-MomChantal FergusonAnnemieke M Aartsma-Rus
Published in: Nucleic acid therapeutics (2023)
Keyphrases
  • drug administration
  • amyotrophic lateral sclerosis
  • human health
  • risk assessment
  • combination therapy
  • bone marrow
  • cell therapy
  • smoking cessation