Cost-effectiveness of Voretigene Neparvovec-rzyl vs Standard Care for RPE65-Mediated Inherited Retinal Disease
Author:
Affiliation:
1. Medicus Economics LLC, Boston, Massachusetts
2. Spark Therapeutics Inc, Philadelphia, Pennsylvania
3. currently, Clearside Biomedical, Alpharetta, Georgia
Publisher
American Medical Association (AMA)
Subject
Ophthalmology
Link
https://jamanetwork.com/journals/jamaophthalmology/articlepdf/2738410/jamaophthalmology_johnson_2019_oi_190048.pdf
Reference41 articles.
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2. Next generation sequencing-based molecular diagnosis of retinitis pigmentosa: identification of a novel genotype-phenotype correlation and clinical refinements.;Wang;Hum Genet,2014
3. Four novel mutations in the RPE65 gene in patients with Leber congenital amaurosis.;Simovich;Hum Mutat,2001
4. Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis.;Morimura;Proc Natl Acad Sci U S A,1998
5. Genetics and phenotypes of RPE65 mutations in inherited retinal degeneration.;Thompson;Invest Ophthalmol Vis Sci,2000
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