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1. Modulator treatments for cystic fibrosis: effectiveness and value

Author(s):

Tice, Jeffrey A., author
Chapman, Rick M., author
Seidner, Matt, author
Pearson, Steven D., author
Rind, David M., author

Publication:

[Boston, Massachusetts] : Institute for Clinical and Economic Review, April 27, 2020

Subject(s):

Cystic Fibrosis -- drug therapy
Cystic Fibrosis Transmembrane Conductance Regulator -- drug effects
United States

2. Spinraza and Zolgensma for spinal muscular atrophy: effectiveness and value : final evidence report

Author(s):

Ellis, Alexandra, author
Mickle, Kristin, author
Herron-Smith, Serina, author
Kumar, Varun M., author
Cianciolo, Laura, author
Seidner, Matt, author
Rind, David, author
Pearson, Steven, author
Thokala, P. (Praveen), author
Stevenson, M. (Matt), author

Publication:

[Boston, Massachusetts] : Institute for Clinical and Economic Review, May 24, 2019

Subject(s):

Genetic Therapy
Muscular Atrophy, Spinal -- drug therapy
United States