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1. Is There a ‘Right to Try’ Experimental Therapies? Ethical Criteria for Selecting Patients With Spinal Muscular Atrophy to Receive Nusinersen in an Expanded Access Program.

Author

Jecker, Nancy S.

Subjects
*NUCLEOTIDES, *ETHICS, *MEDICAL ethics, *SPINAL muscular atrophy, *INVESTIGATIONAL drugs, *THERAPEUTICS
Abstract

The article discusses expanded access programs describing the usage of experimental therapy that benefits research participants. Topics discussed information on Food and Drug Administration's approval of nusinersen for the treatment of spinal muscular atrophy (SMA), justification for patient selection criteria on the bases of utilitarian and deontological arguments, and incremental improvement in well-being.

Published
2017
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