RARE DISEASE
Central retinal artery occlusion
Central retinal artery occlusion
Central retinal artery occlusion
Synonyms: CRAO
Synonyms: CRAO
Synonyms: CRAO
Drug discovery
2
drugs
With orphan designations
Overview
Central retinal artery occlusion (CRAO) is an ophthalmic emergency characterized by sudden, painless monocular vision loss due to retinal ischemia, typically caused by thromboembolism from carotid/cardiac sources or vasculitis (e.g., giant cell arteritis). Diagnosis relies on funduscopy findings (pale retina, cherry-red spot, arterial attenuation) and systemic evaluation for embolic sources. Prognosis is poor, with <20% achieving functional visual recovery without timely intervention. Urgent referral to stroke centers is critical due to elevated cerebrovascular risk [1][4][9].
Therapies
Acute phase: Ocular massage, intraocular pressure reduction (topical timolol, IV acetazolamide), and hyperbaric oxygen (if <12 hours) [3][9][17].
Thrombolytics: IV or intra-arterial tPA within 4.5–6 hours may improve outcomes but lack robust evidence [7][9][12].
Secondary prevention: Antiplatelet therapy, statins, and management of vascular risk factors [4][6][9].
Categories: rare ophthalmic disorders
Research Papers
737 drug discovery papers about Central retinal artery occlusion, with 3 first-in-class and 2 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
737 drug discovery papers about Central retinal artery occlusion, with 3 first-in-class and 2 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-08-17 | Association of Metabolic Syndrome and Its Components with Retinal Artery Occlusion: An Epidemiologic and Genetic Analysis.
To examine the association between MetS, its five components (central obesity, hypertension, hyperglycemia, dyslipidemia, and hypertriglyceridemia) and RAO, and to assess whether genetic susceptibility to key components of the MetS (hypertension, hyperglycemia) interacts with these associations. Based on the UK Biobank, Cox proportional hazards regression models were applied to assess the associations between MetS, its individual components, and the incidence of RAO. Restricted cubic spline analysis was applied to determine non-linear trends in their associations. Polygenic risk scores of hypertension and diabetes were further used to assess the genetic interactions of MetS components with RAO. A total of 361,578 participants with a mean age of 55.94 years were included in the analysis. The risk of RAO was significantly higher in individuals with MetS (HR: 1.59, 95% CI: 1.05-2.42), as well as in those with its key components: hypertension (HR: 3.06, 95% CI: 1.52-6.16) and hyperglycemia (HR: 2.70, 95% CI: 1.27-3.83). Genetic analysis revealed that MetS was associated with a significantly increased risk of RAO in individuals with a higher genetic predisposition to T2DM (HR: 3.20, 95% CI: 2.23-4.17). MetS, especially its key components hypertension and hyperglycemia, increased the risks of RAO onset. Genetic susceptibility to T2DM also increased RAO risk in MetS patients. These findings indicate that MetS may serve as a valuable marker for the secondary prevention of RAO, and stringent management of hyperglycemia and hypertension could contribute to a reduced risk of RAO.
2026-08-14 | Impact of an eye-stroke protocol with non-mydriatic ocular imaging in an emergency department.
The diagnosis of acute central retinal artery occlusion (CRAO) and branch retinal artery occlusion (BRAO) is often delayed or missed in emergency departments (EDs) because of limited ocular funduscopic skills and lack of immediate ophthalmology access. We evaluated the clinical impact of our Eye-Stroke protocol using non-mydriatic ocular imaging (non-mydriatic fundus photography and optical coherence tomography [NMFP-OCT]) in our general ED with remote interpretation by ophthalmology on the time to diagnosis and emergent management of acute CRAO/BRAO. Prospective consecutive series of 100 acute CRAO/BRAOs seen within 1 week of vision loss between June 2023 and April 2026 with NMFP-OCT in our ED. Among 100 CRAO/BRAO eyes seen within 1 week of onset, 16 (16%) had vision loss within 4.5 h, 49 (49%) between 4.5 and 24 h, 35 (35%) between 24 h and 1 week (median times from presentation to NMFP-OCT 28.5 min [IQR, 17.75-57.5 min; range, 10-330 min], 96 min [IQR, 51-180 min; range; 9-317 min], and 134 min [IQR, 96-187 min; range, 30-337 min], respectively). The diagnosis of CRAO/BRAO was made from color photographs and OCT in 77/100 eyes, from OCT only in 19/100 eyes, and 3/100 eyes had uninterpretable imaging. 7/16 eyes presenting within 4.5 h of vision loss received intravenous thrombolysis (median door-to-needle time, 58 min [IQR, 46-78.5 min; range, 41-181 min]). The majority of the patients evaluated within 4.5 h were self-referred to our ED, whereas the majority of the patients presenting later were referred by outside providers or transferred from other EDs. Stroke workup found a major cause of CRAO/BRAO in 72%, and 17/94 (18%) had concurrent cerebral infarctions on brain magnetic resonance imaging (MRI). Despite our Eye-Stroke protocol, facilitated by NFMP-OCT in our ED, only 16% of acute CRAO/BRAO eyes were diagnosed early enough to be considered for intravenous (IV) thrombolysis, which was administered to only 43% of those eligible. Rapid workup found a major cause of CRAO/BRAO in 72%, and 18% had concurrent cerebral infarctions on MRI. The main barrier to delayed diagnosis/care was transfer from other institutions/providers, suggesting that wide deployment of NMFP-OCT for remote diagnosis and treatment via existing telestroke networks is optimal for reducing time to diagnosis, avoiding transfers, and improving patient outcomes.
2026-08-14 | Low-dose intravenous alteplase for acute retinal artery occlusion: A multicentre retrospective study.
Central retinal artery occlusion (CRAO) is an ophthalmic emergency with poor visual prognosis. Although intravenous thrombolysis (IVT) within 4.5 h may be beneficial, the effectiveness and safety of low-dose alteplase remain uncertain. We conducted a retrospective study at three centres in Japan, enrolling patients who presented within 24 h of onset with CRAO or macula-involving branch retinal artery occlusion (BRAO) between June 2021 and September 2024. Patients were analysed if they had baseline best-corrected visual acuity (BCVA) < 20/400, clearly defined symptom onset, absence of proliferative retinopathy or other retinal vascular diseases, and 30-day visual outcome data. Patients were grouped by treatment with IVT using alteplase at 0.6 mg/kg within 4.5 h or non-IVT management. The primary outcome was 30-day BCVA ≥ 20/100; secondary outcome included change in BCVA (logarithm of the minimum angle of resolution [logMAR]); and safety outcomes included intracranial hemorrhage (ICH). Sixteen of 41 registered patients were analysed (70.1 ± 12.6 years; 4 women; 13 had CRAO; 9 received IVT). The primary outcome was achieved in 22.2% (2/9) of the IVT group versus 0% (0/7) of the non-IVT group (p = 0.475). Improvement in logMAR was greater in the IVT than the non-IVT group (median difference 0.45 [95% confidence interval, 0.18-1.20]; p = 0.023). No symptomatic ICH occurred; one IVT-treated patient had asymptomatic ICH. IVT using low-dose alteplase within 4.5 h of CRAO (or macula-involving BRAO) onset may be associated with greater visual improvement without apparent safety concerns, although this requires confirmation by larger prospective studies.
2026-08-10 | Combined Central Retinal Artery Occlusion, Central Retinal Vein Occlusion, and Anterior Ischemic Optic Neuropathy Following Herpes Zoster Ophthalmicus: A Case Report
International audience
2026-07-29 | Sight-saving outcome with thrombolysis in central retinal artery occlusion.
'Time is brain' is an important concept in ischaemic brain stroke, where expeditious intervention is essential for the preservation of neural tissue. Therapeutic strategies involve the prompt administration of thrombolytic agents and in select circumstances the implementation of mechanical thrombectomy. Central retinal artery occlusion (CRAO) constitutes an ophthalmological emergency and is a form of ischaemic stroke, frequently resulting in profound and irreversible visual impairment. There are currently no universally accepted guidelines delineating the optimal therapeutic time window. In this report, we present the case of a man in his 60s who developed acute, painless monocular vision loss, was rapidly diagnosed with CRAO and subsequently received intravenous tenecteplase and showed near-complete visual recovery within 1 week. This case demonstrates the importance of early interdisciplinary collaboration between ophthalmology and stroke teams and the need for establishment of standardised clinical pathways for the management of CRAO.
2026-08-17 | Association of Metabolic Syndrome and Its Components with Retinal Artery Occlusion: An Epidemiologic and Genetic Analysis.
To examine the association between MetS, its five components (central obesity, hypertension, hyperglycemia, dyslipidemia, and hypertriglyceridemia) and RAO, and to assess whether genetic susceptibility to key components of the MetS (hypertension, hyperglycemia) interacts with these associations. Based on the UK Biobank, Cox proportional hazards regression models were applied to assess the associations between MetS, its individual components, and the incidence of RAO. Restricted cubic spline analysis was applied to determine non-linear trends in their associations. Polygenic risk scores of hypertension and diabetes were further used to assess the genetic interactions of MetS components with RAO. A total of 361,578 participants with a mean age of 55.94 years were included in the analysis. The risk of RAO was significantly higher in individuals with MetS (HR: 1.59, 95% CI: 1.05-2.42), as well as in those with its key components: hypertension (HR: 3.06, 95% CI: 1.52-6.16) and hyperglycemia (HR: 2.70, 95% CI: 1.27-3.83). Genetic analysis revealed that MetS was associated with a significantly increased risk of RAO in individuals with a higher genetic predisposition to T2DM (HR: 3.20, 95% CI: 2.23-4.17). MetS, especially its key components hypertension and hyperglycemia, increased the risks of RAO onset. Genetic susceptibility to T2DM also increased RAO risk in MetS patients. These findings indicate that MetS may serve as a valuable marker for the secondary prevention of RAO, and stringent management of hyperglycemia and hypertension could contribute to a reduced risk of RAO.
2026-08-14 | Impact of an eye-stroke protocol with non-mydriatic ocular imaging in an emergency department.
The diagnosis of acute central retinal artery occlusion (CRAO) and branch retinal artery occlusion (BRAO) is often delayed or missed in emergency departments (EDs) because of limited ocular funduscopic skills and lack of immediate ophthalmology access. We evaluated the clinical impact of our Eye-Stroke protocol using non-mydriatic ocular imaging (non-mydriatic fundus photography and optical coherence tomography [NMFP-OCT]) in our general ED with remote interpretation by ophthalmology on the time to diagnosis and emergent management of acute CRAO/BRAO. Prospective consecutive series of 100 acute CRAO/BRAOs seen within 1 week of vision loss between June 2023 and April 2026 with NMFP-OCT in our ED. Among 100 CRAO/BRAO eyes seen within 1 week of onset, 16 (16%) had vision loss within 4.5 h, 49 (49%) between 4.5 and 24 h, 35 (35%) between 24 h and 1 week (median times from presentation to NMFP-OCT 28.5 min [IQR, 17.75-57.5 min; range, 10-330 min], 96 min [IQR, 51-180 min; range; 9-317 min], and 134 min [IQR, 96-187 min; range, 30-337 min], respectively). The diagnosis of CRAO/BRAO was made from color photographs and OCT in 77/100 eyes, from OCT only in 19/100 eyes, and 3/100 eyes had uninterpretable imaging. 7/16 eyes presenting within 4.5 h of vision loss received intravenous thrombolysis (median door-to-needle time, 58 min [IQR, 46-78.5 min; range, 41-181 min]). The majority of the patients evaluated within 4.5 h were self-referred to our ED, whereas the majority of the patients presenting later were referred by outside providers or transferred from other EDs. Stroke workup found a major cause of CRAO/BRAO in 72%, and 17/94 (18%) had concurrent cerebral infarctions on brain magnetic resonance imaging (MRI). Despite our Eye-Stroke protocol, facilitated by NFMP-OCT in our ED, only 16% of acute CRAO/BRAO eyes were diagnosed early enough to be considered for intravenous (IV) thrombolysis, which was administered to only 43% of those eligible. Rapid workup found a major cause of CRAO/BRAO in 72%, and 18% had concurrent cerebral infarctions on MRI. The main barrier to delayed diagnosis/care was transfer from other institutions/providers, suggesting that wide deployment of NMFP-OCT for remote diagnosis and treatment via existing telestroke networks is optimal for reducing time to diagnosis, avoiding transfers, and improving patient outcomes.
2026-08-14 | Low-dose intravenous alteplase for acute retinal artery occlusion: A multicentre retrospective study.
Central retinal artery occlusion (CRAO) is an ophthalmic emergency with poor visual prognosis. Although intravenous thrombolysis (IVT) within 4.5 h may be beneficial, the effectiveness and safety of low-dose alteplase remain uncertain. We conducted a retrospective study at three centres in Japan, enrolling patients who presented within 24 h of onset with CRAO or macula-involving branch retinal artery occlusion (BRAO) between June 2021 and September 2024. Patients were analysed if they had baseline best-corrected visual acuity (BCVA) < 20/400, clearly defined symptom onset, absence of proliferative retinopathy or other retinal vascular diseases, and 30-day visual outcome data. Patients were grouped by treatment with IVT using alteplase at 0.6 mg/kg within 4.5 h or non-IVT management. The primary outcome was 30-day BCVA ≥ 20/100; secondary outcome included change in BCVA (logarithm of the minimum angle of resolution [logMAR]); and safety outcomes included intracranial hemorrhage (ICH). Sixteen of 41 registered patients were analysed (70.1 ± 12.6 years; 4 women; 13 had CRAO; 9 received IVT). The primary outcome was achieved in 22.2% (2/9) of the IVT group versus 0% (0/7) of the non-IVT group (p = 0.475). Improvement in logMAR was greater in the IVT than the non-IVT group (median difference 0.45 [95% confidence interval, 0.18-1.20]; p = 0.023). No symptomatic ICH occurred; one IVT-treated patient had asymptomatic ICH. IVT using low-dose alteplase within 4.5 h of CRAO (or macula-involving BRAO) onset may be associated with greater visual improvement without apparent safety concerns, although this requires confirmation by larger prospective studies.
2026-08-10 | Combined Central Retinal Artery Occlusion, Central Retinal Vein Occlusion, and Anterior Ischemic Optic Neuropathy Following Herpes Zoster Ophthalmicus: A Case Report
International audience
2026-07-29 | Sight-saving outcome with thrombolysis in central retinal artery occlusion.
'Time is brain' is an important concept in ischaemic brain stroke, where expeditious intervention is essential for the preservation of neural tissue. Therapeutic strategies involve the prompt administration of thrombolytic agents and in select circumstances the implementation of mechanical thrombectomy. Central retinal artery occlusion (CRAO) constitutes an ophthalmological emergency and is a form of ischaemic stroke, frequently resulting in profound and irreversible visual impairment. There are currently no universally accepted guidelines delineating the optimal therapeutic time window. In this report, we present the case of a man in his 60s who developed acute, painless monocular vision loss, was rapidly diagnosed with CRAO and subsequently received intravenous tenecteplase and showed near-complete visual recovery within 1 week. This case demonstrates the importance of early interdisciplinary collaboration between ophthalmology and stroke teams and the need for establishment of standardised clinical pathways for the management of CRAO.
Access all drug discovery papers and probability of success in trials forecasts:
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Drug Discovery Landscape
2 orphan drug designations for Central retinal artery occlusion.
2 orphan drug designations for Central retinal artery occlusion.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Sodium 4-amino-3- [6-( 4-fluoro-2-methylphenyl)pyridine-3-ylazo] naphthalene-1-sulfonate dihydrate | small molecules | FDA | 2022-05-03 | — | Kyoto Drug Discovery & Development Co., Ltd. |
mesencephalic, astrocyte-derived neurotrophic factor | proteins | FDA | 2015-09-10 | — | Amarantus BioScience Holdings, Inc. |
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