Last Posted: Jul 14, 2018
- FDA Issues Set of Gene Therapy Draft Guidances
Global Genes, July 11, 2018 - Gene edited monkeys offer hope for heart disease patients
J Kaiser, Science, July 9, 2018 - Are Payers Ready To Address The Financial Challenges Associated With Gene Therapy?
Health Affairs Blog, June 28, 2018 - Genetic Therapies for Sickle Cell Disease.
Jayavaradhan Rajeswari et al. Pediatric clinics of North America 2018 Jun 65(3) 465-480 - New Treatment for Phenylketonuria (PKU) Clears Brain Fog
R Lewis, PLOS Blogs, June 14, 2018 - Sickle Cell Anemia and Its Phenotypes.
Williams Thomas N et al. Annual review of genomics and human genetics 2018 Apr - Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug.
Ferrua Francesca et al. Human gene therapy 2017 Nov 28(11) 972-981 - With tantalizing early results, Sarepta’s gene therapy for Duchenne raises hopes for ‘real change’
A Feuerstein, StatNews, June 19, 2018 - Gene therapy is saving children’s lives—but screening to discover who needs it is lagging behind
E Mullin, Tech Review, May 31, 2018 - Scott Gottlieb Expects the FDA to Approve 40 Gene Therapies by 2022
F Holme, Frontline Genomics, June 8, 2018
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