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About 633 results

ALLMedicine™ Stargardt Disease Center

Research & Reviews  243 results

A Stargardt disease-like phenotype in GAS8-related primary ciliary dyskinesia.
https://doi.org/10.1080/13816810.2022.2090012
Ophthalmic Genetics; Khan AO

Jun 24th, 2022 - Ciliopathies are broadly classified as non-motile or motile (primary ciliary dyskinesia). Early-onset retinal dystrophy is common in non-motile ciliopathy, but retinal dystrophy is not considered a feature of primary ciliary dyskinesia. The subjec...

Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects Lesion(s) in Adolescent Subjects With STGD1
https://clinicaltrials.gov/ct2/show/NCT05244304

May 6th, 2022 - Approximately 60 subjects will be enrolled in this study. Subjects will be assigned to study drug (tinlarebant 5 mg/placebo) with treatment period of upto 24 months with 28 days of follow-up.

ABCA4 c.859-25A>G, a Frequent Palestinian Founder Mutation Affecting the Intron 7 Branc...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9055564
Investigative Ophthalmology & Visual Science; Corradi Z, Salameh M et. al.

Apr 28th, 2022 - The effect of noncoding variants is often unknown in the absence of functional assays. Here, we characterized an ABCA4 intron 7 variant, c.859-25A>G, identified in Palestinian probands with Stargardt disease (STGD) or cone-rod dystrophy (CRD). We ...

Prevalence, multimodal imaging and genotype-phenotype assessment of trauma related subr...
https://doi.org/10.1177/11206721221093986
European Journal of Ophthalmology; Jimenez-Rolando B, Garcia-Sandoval B et. al.

Apr 16th, 2022 - Stargardt disease produces lipofuscin accumulation predisposing to subretinal fibrosis (SRFib) after ocular trauma. Noninvasive imaging techniques allow in vivo assessment. The purpose of this study is to determine the prevalence of SRFib in a coh...

Rare missense mutations in ABCA7 might increase Alzheimer's disease risk by plasma memb...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8973822
Acta Neuropathologica Communications; Bossaerts L, Hendrickx Van de Craen E et. al.

Apr 2nd, 2022 - The adenosine triphosphate-binding cassette subfamily A member 7 gene (ABCA7) is associated with Alzheimer's disease (AD) in large genome-wide association studies. Targeted sequencing of ABCA7 suggests a role for rare premature termination codon (...

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Clinicaltrials.gov  7 results

Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects Lesion(s) in Adolescent Subjects With STGD1
https://clinicaltrials.gov/ct2/show/NCT05244304

May 6th, 2022 - Approximately 60 subjects will be enrolled in this study. Subjects will be assigned to study drug (tinlarebant 5 mg/placebo) with treatment period of upto 24 months with 28 days of follow-up.

Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease
https://clinicaltrials.gov/ct2/show/NCT02402660

Mar 3rd, 2022 - This study evaluates the effects of orally-administered ALK-001 on the progression of Stargardt disease (ABCA4-related). Stargardt disease is a rare genetic disorder that leads to damage to the retina and results in legal blindness. Stargardt dise...

Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)
https://clinicaltrials.gov/ct2/show/NCT04239625

Jul 21st, 2021 - The purpose of this open-label, multicenter study is to determine the long-term safety, pharmacokinetics and effects of ALK-001 (C20-D3-retinyl acetate) on the progression of Stargardt disease. This study is an extension of NCT02402660 and enrolls...

Study of STG-001 in Subjects With Stargardt Disease
https://clinicaltrials.gov/ct2/show/NCT04489511

Apr 27th, 2021 - This is an open-label, multicenter, active treatment study in approximately 12 subjects aged 18 to 55 years (inclusive) with STGD1, genotyped with a minimum of two ABCA4 gene mutations. Treatment course of STG-001 at Dose 1 (Cohort 1) or Dose 2 (C...

Stem Cells Therapy in Degenerative Diseases of the Retina
https://clinicaltrials.gov/ct2/show/NCT03772938

Dec 17th, 2018 - Degenerative diseases of the retina are challenging for ophthalmologists. This is a common term that covers heterogenous group of diseases, f.e. retinitis pigmentosa, Stargardt disease, Best's disease or age related macular degeneration. Undetermi...

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News  3 results

Unsafe Ocular Stem Cell Treatments Prevalent
https://www.medscape.com/viewarticle/899932

Jul 27th, 2018 - VANCOUVER, Canada — Experimental stem cell therapies for eye conditions are being offered at dozens of unregulated clinics in the United States, and some of these procedures are harmful, researchers report. Clinicians should warn their patients ag...

Gene Therapy for Retinal Diseases Is Within Sight
https://www.medscape.com/viewarticle/824467

May 7th, 2014 - Advances in Vitreoretinal Disease Management Many vitreoretinal pathologies have witnessed impressive advances in their management over the past decade. Most of these advances have involved pharmacologic innovation aimed at controlling exudative d...

Gene Therapy for Retinal Diseases Is Within Sight
https://www.staging.medscape.com/viewarticle/824467

May 7th, 2014 - Advances in Vitreoretinal Disease Management Many vitreoretinal pathologies have witnessed impressive advances in their management over the past decade. Most of these advances have involved pharmacologic innovation aimed at controlling exudative d...

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