Achromatopsia
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CLINICAL_TRIALS research · 2025-12-10Clinical Trial Update: Natural History Study of Inherited Retinal Diseases
A natural history study of inherited retinal diseases sponsored by Zhongmou Therapeutics is currently recruiting. The study will examine ret…
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CLINICAL_TRIALS trial_result · 2025-09-12Clinical Trial Update: PBA Use for Treatment of ATF6-/- Patients [EARLY_Phase 1]
The EARLY Phase 1 clinical trial evaluating phenylbutyrate for ATF6-/- patients with achromatopsia is currently suspended, in a study sponso…
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CLINICAL_TRIALS trial_result · 2025-06-11Clinical Trial Update: Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3
MeiraGTx reported a long-term follow-up for a CNGB3/CNGA3 gene therapy in Achromatopsia; the trial was terminated and results were posted in…
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CLINICAL_TRIALS trial_result · 2024-04-18Clinical Trial Update: Safety and Efficacy of rAAV.hCNGA3 Gene Therapy in Patients With CNGA3-linked Achromatopsia [Phase 1, Phase 2]
Phase 1/2 clinical trial evaluating safety and efficacy of rAAV.hCNGA3 gene therapy for CNGA3-linked Achromatopsia; trial status is active b…
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CLINICAL_TRIALS trial_result · 2023-03-08Clinical Trial Update: Gene Therapy for Achromatopsia (CNGB3) [Phase 1, Phase 2]
Phase 1/2 trial of gene therapy for achromatopsia (CNGB3) completed with results posted in 2023.
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CLINICAL_TRIALS trial_result · 2022-07-22Clinical Trial Update: Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial) [Phase 1, Phase 2]
Aacygene therapy trial (AAV) for CNGB3-related achromatopsia is ongoing in Phase 1/2 to assess safety and efficacy.
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CLINICAL_TRIALS trial_result · 2022-07-22Clinical Trial Update: Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial) [Phase 1, Phase 2]
A clinical trial update for an AAV gene therapy targeting CNGA3 achromatopsia (A Clarity Clinical Trial) across Phase 1 and Phase 2; status …
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CLINICAL_TRIALS trial_result · 2022-02-11Clinical Trial Update: Gene Therapy for Achromatopsia (CNGA3) [Phase 1, Phase 2]
Clinical trial update for a gene therapy targeting CNGA3 in achromatopsia completed with results posted in 2022.
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CLINICAL_TRIALS research · 2017-10-13Clinical Trial Update: Clinical and Genetic Characterization of Individuals With Achromatopsia
This clinical trial update summarizes the clinical and genetic characterization of individuals with achromatopsia, describing baseline pheno…
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CLINICAL_TRIALS trial_result · 2014-02-25Clinical Trial Update: CNTF Implants for CNGB3 Achromatopsia [Phase 1, Phase 2]
Phase 1/2 clinical trial evaluating CNTF implants for CNGB3-related achromatopsia has completed with results posted in 2014.