Gene Therapy for Choroideremia-Progress and Remaining Questions. Duncan Jacque L et al. JAMA ophthalmology 2019 Aug

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Last Posted: Sep 01, 2019

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• 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
• This Drug Will Save Children’s Lives. It Costs $2 Million.
  NY Times editorial board, August 13, 2019
• Progress and challenges of gene therapy for Pompe disease.
  Ronzitti Giuseppe et al. Annals of translational medicine 2019 Jul 7(13) 287

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