Ambylopia treatment study to compare the effectiveness of binocular game play versus patching in children ages 5 to < 17 years of age.
The purpose of this study will be to collect a skin biopsy (skin tissue) or blood sample from each participating patient with retinal degeneration, such as Best’s disease or age-related macular degeneration (AMD), including geographic atrophy (GA). Each skin biopsy or blood sample will be taken to the laboratory of the investigators where skin epithelial cells called fibroblasts, will be induced to make “pluripotent stem cells” or “iPSCs”. These iPSCs will be differentiated into retina epithelial cells which will be tested for their ability to function as new retinal cells in animal models of retinal degeneration. Because these special cells are individualized or patient-specific, they will not be rejected by that individual patient, and thereby represent the first step in a potential future treatment for retinal disease. Cells will also be processed at the New York Stem Cell Foundation IPSC core facility using the procedure described above. The Medical University of South Carolina (MUSC) and New York Stem Cell Foundation (NYSCF) will enter into a material transfer agreement (MTA) initiated by the MUSC Foundation for Research Development. Once MTAs are initiated, samples will be coded at MUSC. Coded samples will be sent via dry ice to ensure full competency to the NYSCF. Samples will remain coded at MUSC and will not be released to the NYSCF. Fibroblasts will be reprogrammed using the method described above and sent back to MUSC (as iPS cells) for the research purposes of this study. Cells will only be identifiable to the NYSCF by disease, sex, and gender. Once cells are received by the NYSCF they receive a number bar code for further processing and are in no way traceable to the patient by the NYSCF. Reprogrammed cells will be stored at the NYSCF biorepository but only shared with the knowledge of MUSC. These samples will be used for the purposes of this study. Any other use will be evaluated by the investigators and the MUSC Foundation for Research Development and the appropriate action taken. Once samples are processed that will not be withdrawn from the biorepository. The NYSCF biorepository is a secure facility with only authorized personnel having access to the facility
This study is to compare the effect of an investigational drug (Lampalizumab) in an injection to the eye every 4 to 6 weeks compared to a sham (false ) injection on the effects of Geographic Atrophy secondary to patients with Age Related Macular Degeneration. This study will last for 2 years. The study will involve blood draws and numerous ophthalmic exams.
Children aged 6-11-years-old with Cystic Fibrosis who are homozygous for the F508del-CFTR are welcome to volunteer for this study.
This study is being done to learn more about the safety and tolerability of taking lumacaftor with ivacaftor.
Ivacaftor, at a different dose, is approved for some patients aged 6 and older in the US and certain other countries. Ivacaftor is marketed in the US under the trade name Kalydeco. Ivacaftor is experimental in patients with CF that are homozygous for the F508del CFTR mutation.
This research study will test whether or not using an investigational drug called OMS302 during pediatric cataract surgery helps keep the pupil dilated (open) and reduces eye pain after surgery. OMS302 (Omidria™ phenylephrine and ketorolac injection 1%/0.3%) has been approved by United States Food and Drug Administration for use in adults to keep the pupil dilated during surgery and reduce eye pain after the surgery. The use of Omidria™ in children has not been FDA-approved. This study is being done to help gain FDA approval for this drug’s use during cataract surgeries on children up to 3 years old.
The purpose of this study is looking at the use of a treatment for slowing the progression (worsening) of Keratoconus
The purpose of this project is to develop and test preliminary reliability of a newly developed pediatric tool, the Pediatric Sensory Modality Assessment and Rehabilitation Techniques (SMART), which will measure cognitive awareness for children with severe brain damage. Seven children, between the ages of 3-12 years, with physician-documented severe brain damage and considered medically stable are needed for this study. Recruitment flyers will be disseminated at MUSC and the greater Charleston area community to recruit parents/legal guardians of children with severe brain damage. Once parental/legal guardian and physician consents are in place, participants will be evaluated using the Pediatric SMART 5 times within 10 days. The Pediatric SMART is made up of 5 domains that are olfactory, visual, auditory and vestibular, gustatory, and tactile. Test administration requires approximately 1 hour and can be completed in settings convenient for parents/legal guardians. The potential benefit to study participants is that the findings from the Pediatric SMART may identify sensory and motor strengths of participants. Knowledge of these strengths may enhance current rehabilitation and treatment plans, which may lead to functional improvements; although, this cannot be guaranteed. It is a goal of this study to evaluate the preliminary Pediatric SMART reliability. Once reliability has been substantiated for the Pediatric SMART in further and future study, future children with severe brain injury, being evaluated with the Pediatric SMART, may have rehabilitation and treatments opportunities that are better informed, leading to greater improvement in functional and participatory outcomes.
This study is intended to compare the accuracy of the visual acuity measurement done with a new projected eyechart method when compared to the gold standard (Marco Guyton-Minkowski PAM) before surgery and Snellen wallchart acuity after cataract surgery.
This research is being done with children who have a condition called intermittent exotropia. Intermittent exotropia is the medical term used when the eyes turn out some of the time and are straight at other times. Intermittent exotropia is one of the most common types of eye misalignment in children.
Intermittent exotropia is often treated with surgery on the eye muscles to make the eyes straight again. There are different ways to do this surgery to straighten the eyes. One way is to operate on two muscles on one eye. Another way is to operate on one muscle on each eye. Both ways of doing the surgery work well but we do not know if one way is better than the other. This is the reason why the study is being done.