Last Posted: Sep 10, 2019
- First gene therapy for β-thalassemia approved
C Harrison, Nature Biotechnology, September 9, 2019 - Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia
P Rio et al, Nature Medicine, September 9, 2019 - Gene Therapy for Choroideremia-Progress and Remaining Questions.
Duncan Jacque L et al. JAMA ophthalmology 2019 Aug - Important Considerations in Modeling the Cost-Effectiveness for the First Food and Drug Administration-Approved Gene Therapy and Implications for Future One-Time Therapies.
Buessing Marric et al. Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research 2019 Aug 22(8) 970-971 - Long QT Syndrome: Genetics and Future Perspective.
Wallace Eimear et al. Pediatric cardiology 2019 Aug - Mom says she'll fight for world's most expensive drug for daughter, costs $2 million
AJC, August 22, 2019 - Emerging non-genetic therapies for sickle cell disease.
Carden Marcus A et al. Haematologica 2019 Aug - Analytic Considerations in Applying a General Economic Evaluation Reference Case to Gene Therapy.
Drummond Michael F et al. Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research 2019 22(6) 661-668 - Innovative Therapies for Cystic Fibrosis: The Road from Treatment to Cure.
Cabrini Giulio et al. Molecular diagnosis & therapy 2019 23(2) 263-279 - Recent progress in translational cystic fibrosis research using precision medicine strategies.
Cholon Deborah M et al. Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society 2018 17(2S) S52-S60
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