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Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium
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scholarly article
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PubMed
review article
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stated in
Europe PubMed Central
title
Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium
(English)
1 reference
stated in
PubMed
main subject
retinal degeneration
1 reference
based on heuristic
inferred from title
author
Alessandro Iannaccone
object named as
A. Iannaccone
series ordinal
9
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B Falsini
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Deniz Dalkara
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ORCID Public Data File 2021
author name string
D. A. Thompson
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1
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R. R. Ali
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E. Banin
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K. E. Branham
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J. G. Flannery
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D. M. Gamm
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W. W. Hauswirth
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J. R. Heckenlively
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K. T. Jayasundera
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N. W. Khan
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R. S. Molday
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M. E. Pennesi
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T. A. Reh
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R. G. Weleber
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D. N. Zacks
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language of work or name
English
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publication date
9 February 2015
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PubMed
published in
Investigative Ophthalmology Visual Science
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PubMed
volume
56
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PubMed
issue
2
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PubMed
page(s)
918-31
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PubMed
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RS-1 Gene Delivery to an Adult Rs1h Knockout Mouse Model Restores ERG b-Wave with Reversal of the Electronegative Waveform of X-Linked Retinoschisis
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Viral-mediated RdCVF and RdCVFL expression protects cone and rod photoreceptors in retinal degeneration
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Gene therapy in patient-specific stem cell lines and a preclinical model of retinitis pigmentosa with membrane frizzled-related protein defects
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EIAV-based retinal gene therapy in the shaker1 mouse model for usher syndrome type 1B: development of UshStat
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Photoreceptor precursors derived from three-dimensional embryonic stem cell cultures integrate and mature within adult degenerate retina.
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Transduction of photoreceptors with equine infectious anemia virus lentiviral vectors: safety and biodistribution of StarGen for Stargardt disease
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Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement
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Optogenetic therapy for retinitis pigmentosa
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Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years
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29 September 2017
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retrieved
29 September 2017
Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Allelic and phenotypic heterogeneity in ABCA4 mutations
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Restoration of cone vision in the CNGA3-/- mouse model of congenital complete lack of cone photoreceptor function
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Genetic reactivation of cone photoreceptors restores visual responses in retinitis pigmentosa
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Animals as models of age-related macular degeneration: an imperfect measure of the truth
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
The molecular basis of human retinal and vitreoretinal diseases
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Generation, purification and transplantation of photoreceptors derived from human induced pluripotent stem cells
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Protective effects of human iPS-derived retinal pigment epithelium cell transplantation in the retinal dystrophic rat
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Gene therapy for red-green colour blindness in adult primates
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Intravitreal delivery of AAV8 retinoschisin results in cell type-specific gene expression and retinal rescue in the Rs1-KO mouse
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Naturally occurring animal models with outer retina phenotypes
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Management of autoimmune retinopathies with immunosuppression
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Prospects for retinal gene replacement therapy.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Elucidating the phenomenon of HESC-derived RPE: anatomy of cell genesis, expansion and retinal transplantation.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
RPE transplantation and its role in retinal disease
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Reduced-illuminance autofluorescence imaging in ABCA4-associated retinal degenerations
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Lentiviral gene replacement therapy of retinas in a mouse model for Usher syndrome type 1B.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Retinal repair by transplantation of photoreceptor precursors
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Efficient generation of retinal progenitor cells from human embryonic stem cells.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
In vivo dynamics of retinal injury and repair in the rhodopsin mutant dog model of human retinitis pigmentosa
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Biodistribution of rAAV vectors following intraocular administration: evidence for the presence and persistence of vector DNA in the optic nerve and in the brain.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Identification and characterization of rod-derived cone viability factor
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
In vitro and in vivo characterization of pigment epithelial cells differentiated from primate embryonic stem cells.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Neural remodeling in retinal degeneration.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Selective transplantation of rods delays cone loss in a retinitis pigmentosa model
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Mutation of a gene encoding a protein with extracellular matrix motifs in Usher syndrome type IIa.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
29 September 2017
Comprehensive registration of DNA sequence variants associated with inherited retinal diseases in Leiden Open Variation Databases
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
2 June 2018
Intravitreal delivery of AAV-NDI1 provides functional benefit in a murine model of Leber hereditary optic neuropathy
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
2 June 2018
Long-term rescue of retinal structure and function by rhodopsin RNA replacement with a single adeno-associated viral vector in P23H RHO transgenic mice.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
2 June 2018
Changes in adeno-associated virus-mediated gene delivery in retinal degeneration
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
2 June 2018
RNA interference-mediated suppression and replacement of human rhodopsin in vivo
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
2 June 2018
A single, abbreviated RPGR-ORF15 variant reconstitutes RPGR function in vivo
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
2 June 2018
Submicrovolt flicker electroretinogram: cycle-by-cycle recording of multiple harmonics with statistical estimation of measurement uncertainty.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
2 June 2018
Standardized full-field electroretinography. Normal values and their variation with age
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
2 June 2018
The special case of gene therapy pricing
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
27 November 2018
Diagnostic fundus autofluorescence patterns in achromatopsia.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
27 November 2018
In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
27 November 2018
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
27 November 2018
A novel approach for subretinal implantation of ultrathin substrates containing stem cell-derived retinal pigment epithelium monolayer
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
27 November 2018
Production of ELOVL4 transgenic pigs: a large animal model for Stargardt-like macular degeneration.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
27 November 2018
ISCEV standard for clinical electro-oculography (2010 update).
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
27 November 2018
Test-retest reliability and inter-ocular symmetry of multi-focal electroretinography in Stargardt disease
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
27 November 2018
Generation of a transgenic rabbit model of retinal degeneration
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
27 November 2018
Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of x-linked juvenile retinoschisis
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
27 November 2018
Quantifying rod photoreceptor-mediated vision in retinal degenerations: dark-adapted thresholds as outcome measures
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
27 November 2018
Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
27 November 2018
Persistent transgene product in retina, optic nerve and brain after intraocular injection of rAAV.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
27 November 2018
Genetically engineered large animal model for studying cone photoreceptor survival and degeneration in retinitis pigmentosa
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4554258
retrieved
27 November 2018
Oral 9-cis retinoid for childhood blindness due to Leber congenital amaurosis caused by RPE65 or LRAT mutations: an open-label phase 1b trial
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/25667399
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
A new era in medical therapy for retinal degenerative disease?
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/25667399
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
The effect of age on human cone and rod ganzfeld electroretinograms
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/25667399
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
Assessment of foveal cone photoreceptors in Stargardt's macular dystrophy using a small dot detection task
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/25667399
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
Report from the NEI/FDA Ophthalmic Clinical Trial Design and Endpoints Symposium
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/25667399
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
Identifiers
DOI
10.1167/IOVS.14-16049
0 references
PMC publication ID
4554258
1 reference
stated in
PubMed
PubMed publication ID
25667399
1 reference
stated in
PubMed
ResearchGate publication ID
272095323
0 references
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