Disorder of visual system

This early-stage study tested a new oral drug, ASP5502, for primary Sjögren's syndrome, an autoimmune condition causing dry eyes and mouth. It involved 116 healthy adults and patients to check safety and how the body processes the drug. The study was terminated, but results may g…

This study tested a low dose of the drug bevacizumab (Avastin) against standard laser treatment for retinopathy of prematurity, an eye disease that can blind premature infants. Sixteen babies with severe disease were enrolled. The goal was to see if the drug could control the dis…

This early-stage trial tests a radioactive drug called [225Ac]Ac-A9-3408 in 50 adults with melanoma that has spread or can't be removed by surgery. The main goals are to find a safe dose and check for side effects. Participants receive the drug every 6 weeks for up to 6 doses, an…

This pilot study tests whether shining near-infrared light on the eye can reduce macular edema—swelling in the central retina—caused by a retinal vein occlusion. Six participants with this condition will receive 12 light treatments over about five weeks. The main goal is to see i…

This study tests an experimental drug called REGN7041 in 72 people with active noninfectious uveitis, a condition causing inflammation inside the eye. The drug is an antibody designed to calm the immune system. Since this is the first time it's tested in humans, the main goal is …

This trial is testing whether combining two drugs—pembrolizumab and lenvatinib—can help people with metastatic uveal melanoma, a rare eye cancer that has spread to other parts of the body. Participants receive both drugs for up to two years. The study measures how long people liv…

This study tested a new type of artificial lens (TECNIS DEN00V) for people having cataract surgery. It compared the new lens to a standard lens in 243 adults with cataracts. The goal was to see if the new lens improves vision at arm's length (66 cm) while keeping good distance vi…

This early-stage study tests the safety of an experimental drug called RO7669330, given as an injection into the eye, for people with geographic atrophy (GA) due to age-related macular degeneration (AMD). The study involves 27 participants and will monitor side effects and eye he…

This phase 2 trial tests whether a modified Chinese herbal formula, Shenling Baizhu San, can reduce swelling in the retina (macular edema) and improve vision. Adults diagnosed with macular edema take either the herbal granules or a placebo twice daily for 12 weeks. The study meas…

This study tested a drug called BVD-523 (Ulixertinib) in 13 people with advanced uveal melanoma, a rare eye cancer that has spread. The drug works by blocking certain proteins that help cancer grow. The trial was terminated early, so results are limited and it's unclear how well …

This trial tests whether a new type of spectacle lens can slow the progression of myopia (nearsightedness) in children aged 8 to 13. Over 12 months, researchers measure changes in eye length and prescription, comparing results to a historical group that wore standard lenses. The …

This study tests whether using a violet light lamp at home can slow the progression of myopia (nearsightedness) in children. 72 kids aged 5 to 12 will use either a violet light lamp or a regular white light lamp for 12 months. Researchers will measure changes in eye length and vi…

This study tested two different proton therapy schedules for people with large choroidal melanomas (eye cancer) that would normally require removal of the eye. The goal was to see if a gentler radiation schedule could control the tumor and avoid severe side effects, allowing pati…

This study tested whether a daily tablet called MEMOPTIC (containing citicoline, magnesium, and Ginkgo biloba) could help preserve vision in people with open-angle glaucoma when added to their usual eye-pressure-lowering treatments. The trial planned to follow 15 patients for two…

This study tested a new type of artificial lens (enVista Aspire) for people having cataract surgery. The goal was to see if it improves intermediate vision, like seeing a computer screen, better than standard lenses. The trial was stopped early and included 27 adults aged 22 and …

This small pilot study tested whether adding gypenosides (a herbal supplement) to standard steroid treatment could help protect the nerve at the back of the eye in adults having their first episode of acute optic neuritis. Ten participants were enrolled, but the study was stopped…

This study tested whether giving a clot-busting drug (tenecteplase or alteplase) within 4.5 hours of an eye stroke could restore vision. Eye stroke, or central retinal artery occlusion, is a blockage in the eye's main artery that often causes permanent vision loss. The trial aime…

This study compared a new device called miCOR, which uses low energy to break up cataracts, to standard cataract surgery. About 402 adults with cataracts were planned to take part. The study was terminated early, so we don't have full results on whether the new device is better o…

This early-stage trial tested a drug called DS-2325a in 9 adults with Netherton syndrome, a rare genetic condition causing severe skin redness, scaling, and allergies. The study aimed to check safety and whether the drug could help control the disease. However, the trial was term…

This study tested a single injection of avoralstat into the eye for people with diabetic macular edema, a condition that causes vision loss in diabetes. Only 3 adults took part before the study was stopped early. The main goal was to check safety and see if the drug might help re…

This study tested two different combinations of eye drops to lower eye pressure in people with open-angle glaucoma or ocular hypertension. The goal was to see which combination worked better over 90 days. The study was stopped early and included 28 participants.

This study tested an experimental drug called HZN-1116 in 209 people with Sjögren's syndrome, an autoimmune disease causing dry eyes and mouth. The goal was to see if the drug could reduce disease activity and symptoms. The trial was terminated early, so results may not be availa…

This trial tested a device called the CorNeat KPro, an artificial cornea, in 15 legally blind adults who could not get a standard cornea transplant. The goal was to see if the implant is safe and can improve vision. The study was terminated early, so full results are not availabl…

This early-phase trial tested the drug zanubrutinib in just 6 adults with neuromyelitis optica spectrum disorder (NMOSD), a rare condition where the immune system attacks the nerves. Participants took the pill twice daily for a year to see if it could prevent relapses. However, t…

This study tested a drug called secukinumab in adults with active, moderate to severe thyroid eye disease, a condition that causes bulging eyes and discomfort. The trial aimed to see if the drug could reduce eye bulging and inflammation. However, the study was stopped early and o…

This study tested an experimental drug called RC18 in 178 people with neuromyelitis optica spectrum disorders (NMOSD), a rare disease that causes inflammation in the eyes and spinal cord. The goal was to see if RC18 could prevent relapses and slow disability. Participants had to …

This trial tested a computer-based therapy called AmblyoFix for treating lazy eye (amblyopia) in 40 people aged 8 to 19. Participants either used the therapy for 90 minutes daily or wore a standard eye patch. The study was stopped early, and only 4-week data is available, making …

This early-stage study tested a new oral drug called K9 for diabetic macular edema, a condition that causes vision loss in people with diabetes. The trial was stopped early and only enrolled one person. The main goal was to check safety and measure changes in retinal swelling and…

This study looked at the long-term safety and effectiveness of the STREAMLINE® Surgical System in people with open-angle glaucoma. It included 17 patients who had already taken part in an earlier trial of the same device. The goal was to see if the device continued to safely lowe…

This study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …

This study tested whether BHV-7000, taken alongside other medications, could reduce generalized tonic-clonic seizures in people with idiopathic generalized epilepsy. The trial was terminated early and enrolled only 27 adults aged 18-75. The main goal was to see how long it took f…

This study tested a device called the ELITA System to reduce or eliminate nearsightedness (myopia) and astigmatism. It involved 96 adults aged 22 and older who received a lenticule removal procedure in one or both eyes. The trial was terminated early, so the planned results on sa…

This study tested a medicine called spesolimab for people with Netherton syndrome, a rare genetic skin condition causing severe redness and scaling. About 43 people aged 12 and older took part, receiving either the drug or a placebo. The goal was to see if spesolimab could reduce…

This phase II trial tested the drug imatinib in 13 adults with metastatic ocular melanoma, a rare eye cancer that has spread. The goal was to see if imatinib could stop tumor growth for at least 3 months. The trial was terminated early, so the results are limited and it is unclea…

This study compared two antiviral medicines (oral valganciclovir and topical ganciclovir 2%) against a placebo for treating CMV-related inflammation in the front of the eye. The goal was to see which approach better controls the infection and reduces complications like pain, glau…

This study tested a single injection of a gene therapy called GT005 in 98 people with geographic atrophy, an advanced form of dry age-related macular degeneration that causes vision loss. The goal was to see if the treatment could slow the growth of damaged areas in the eye and c…

This Phase 3 trial tested an oral drug called venglustat in 75 adults and children with late-onset Tay-Sachs or Sandhoff disease, rare genetic disorders that cause progressive nerve damage. The drug aimed to lower toxic fat buildup in the brain and slow disease worsening. However…

This study tested a single injection of GT005, a gene therapy, in 255 people with geographic atrophy from dry age-related macular degeneration. The goal was to see if it could slow the growth of blind spots in the eye. The trial was terminated early, so we have less data than hop…

This early-stage trial tested a gene therapy called GT005 for dry age-related macular degeneration (AMD), a leading cause of vision loss. The therapy delivers a working gene to the retina via a single injection. The study enrolled 56 people and focused on safety and dosing, but w…

This early-stage trial tested a modified virus (VSV-IFNbetaTYRP1) in 12 patients with advanced melanoma that had spread or could not be removed by surgery. The virus was designed to infect and kill melanoma cells while also boosting the immune system's attack. The main goals were…

This phase 3 trial tested aflibercept (Eylea) eye injections against placebo in 16 people with macular telangiectasia type 1, a rare condition that causes vision loss. The study aimed to see if aflibercept could reduce swelling in the macula. However, the trial was terminated ear…

This study tested whether intense pulsed light (IPL) can help treat chalazion, a common eyelid lump. Only 4 adults with a fresh chalazion were enrolled, and the trial was stopped early. Because of the very small size, we cannot say if IPL works or is safe for this condition.

This early-phase trial tested a radioactive drug designed to seek out and destroy cancer cells in people with advanced solid tumours that had stopped responding to other treatments. The drug targets a protein called IGF-1R found on many cancer cells. The study aimed to find a saf…

This study tested a medicated ointment called pimecrolimus 0.3% for people with blepharitis, a condition that causes red, irritated eyelids. The trial planned to include 477 participants aged 2 and older, applying the ointment twice daily to the eyelid margins. The main goal was …

This study tested whether a daily dietary supplement called GlaucoCetin could improve vision in people with open-angle glaucoma who already had some vision loss. Only 12 participants were enrolled before the trial was terminated early. The supplement contained a mix of ingredient…

This study tested a gene therapy for retinitis pigmentosa, an inherited eye disease that causes gradual vision loss. The treatment involved injecting a corrected gene under the retina in one eye. The trial included 19 people aged 13 and older with a specific genetic defect. The m…

This early-phase study tested a new drug called KZR-261 in 61 people with advanced solid tumors that had stopped responding to standard treatments. The main goals were to check safety, find the right dose, and see if the drug could shrink tumors. The trial was terminated, but the…

This study tested whether using a virtual reality headset for one-hour sessions could help regenerate damaged optic nerves and improve vision in people with glaucoma or other retinal diseases. The idea came from promising results in rodents. However, the trial was terminated earl…

This study tested whether anti-VEGF injections into the eye could help treat Coats disease, a rare condition that causes abnormal blood vessels and fluid buildup in the retina, potentially leading to blindness. The trial included 18 people with early-stage Coats disease who had n…

This study looked at the long-term safety of an experimental drug called CVL-865 in adults whose focal seizures were not controlled by other medications. About 105 people who completed a previous trial took CVL-865 alongside their usual seizure drugs for up to 57 weeks. The study…

This study tested whether taking vitamin B2 (riboflavin) and getting natural sunlight could strengthen the cornea in people with keratoconus, a condition that causes the cornea to thin and bulge. The approach aimed to be a simpler, cheaper alternative to the standard cross-linkin…

This study looked at whether laser eye surgery (PRK) could improve vision in children aged 2 to 17 with lazy eye (amblyopia) caused by large differences in prescription between eyes or very high nearsightedness, farsightedness, or astigmatism. These children had not improved afte…

This study tested a new surgical technique for macular holes that remained open after standard treatment. The procedure involves moving a thin membrane from the eye to cover the hole. Only 16 people were enrolled, and the study was stopped early, so we don't yet know if this appr…

This study tested a new device (VISCO™360) against a standard laser treatment to lower eye pressure in adults with open-angle glaucoma. Only 29 people took part before the study was stopped early. The goal was to see which method works better and is safer for controlling the dise…

This study tested an oral drug called CX-4945 (silmitasertib) in 21 children with recurrent SHH medulloblastoma, a rare brain tumor. The trial aimed to find the best dose and see if the drug could shrink tumors. However, the study was stopped early, so we don't have clear answers…

This study aimed to compare two surgical devices—the OMNI system and the iStent inject—for lowering eye pressure in people with open-angle glaucoma who already had cataract surgery. The trial was terminated early and only enrolled 10 participants, so results are limited. Both dev…

This early-phase trial tested a new immunotherapy approach for children with DIPG, a rare and aggressive brain stem tumor. After standard radiation and chemotherapy, patients received special vaccines and immune cells designed to attack the tumor. The study was small (11 particip…

This study tested a new drug called SD-101, given directly into the liver through a special pressure-enabled infusion, alone or with standard immunotherapy drugs. It was for adults with a rare eye cancer (uveal melanoma) that had spread mainly to the liver. The main goals were to…

This study tested a special contact lens that slowly releases the glaucoma drug latanoprost, aiming to replace daily eye drops. Only one person enrolled before the study was stopped early. The goal was to check safety and whether the lens could lower eye pressure, but no conclusi…

This study tested the drug perampanel in adults whose seizures did not stop with standard treatments. The goal was to see if it could safely stop seizures and reduce time in the hospital. Only one person enrolled before the study was ended early, so no clear conclusions can be dr…

This study tested whether lorcaserin, when added to current medications, could reduce convulsive seizures in people with Dravet syndrome, a severe form of epilepsy. The trial planned to enroll about 22 participants aged 2 and older, but it was terminated early. The main goal was …

This study tested a new medical device called Supraflow v1.3, which is implanted in the eye to help drain fluid and lower pressure in people with open-angle glaucoma. The trial included 22 patients whose eye pressure was not controlled with medication. However, the study was term…

This phase II trial tested a combination of precise radiation therapy followed by injections of aflibercept (Eylea) in people with uveal melanoma, a rare eye cancer. The goal was to see if this approach could control the tumor while reducing side effects like swelling or damage t…

This phase 3 trial tested the drug miglustat (Zavesca) in 30 infants with Sandhoff or Tay-Sachs diseases, rare genetic disorders that destroy nerve cells. The goal was to see if the drug could reduce hospitalizations, seizures, and feeding problems while improving motor function.…

This Phase 2 study tested an oral drug called danicopan in 365 people with geographic atrophy, an advanced form of dry age-related macular degeneration that causes blind spots. Participants took different doses of danicopan or a placebo for up to 104 weeks to see if it could slow…

This early-stage trial tested a gene therapy called AXO-AAV-GM2 in children with Tay-Sachs or Sandhoff disease, rare and fatal genetic brain disorders. The therapy delivers healthy genes directly into the brain and spinal fluid to try to restore a missing enzyme. The study was te…

This study tested a smartphone app called CRADLE that uses a photo to detect leukocoria (a white glow in the pupil), which can be a sign of retinoblastoma, cataracts, or glaucoma. Researchers planned to compare the app's accuracy to a standard eye exam in children with these cond…

Eye pressure is usually measured with a technique called Goldmann applanation tonometry, which doctors consider the gold standard but which depends on the examiner's skill and offers limited repeatability. Researchers at Duke University are testing automated versions of this meas…

This study tests whether a new tool, the VISIODOL® scale, can help people with visual impairment communicate pain. Researchers will compare pain ratings from this scale with those from a standard numerical scale during heat-induced pain. The study includes healthy volunteers with…

This study tests whether an artificial intelligence program called NeoRetina can accurately detect diabetic retinopathy and diabetic macular edema from simple eye photographs. Researchers will compare the AI's analysis to standard exams by eye doctors. The goal is to see if this …

This study tested whether a virtual reality headset combined with machine learning can measure pupil reactions more reliably than the standard flashlight test for Relative Afferent Pupillary Defect (RAPD). About 71 adults with and without known RAPD took part. Researchers compare…

This study tested a new software called AngioScan, used with an already-approved eye camera, to see if it can take better pictures of the back of the eye. Researchers looked at 150 patients with retinal problems like macular degeneration. The goal was to find out if the software …

This study looked at whether eating corn rich in zeaxanthin (a natural pigment) could increase the density of the macular pigment in the eye, which may help prevent age-related macular degeneration (AMD), a leading cause of blindness. Nine healthy volunteers aged 20-35 ate a dail…

This study tests two experimental drugs (REGN5713 and REGN5715) given alone or together to reduce eye allergy symptoms caused by birch pollen. About 350 adults with birch pollen allergy will receive the drugs or a placebo and then be exposed to pollen in a controlled setting. The…

This trial tests whether OCS-01, a dexamethasone eye drop, can reduce inflammation and pain after cataract surgery. About 117 adults scheduled for cataract removal will receive either the active drop or a placebo once daily. The study measures how well the drop clears eye inflamm…

This study tested a drug called AVP-786 to see if it could safely reduce disinhibition—impulsive or inappropriate behavior—in people with neurodegenerative disorders like Alzheimer's or frontotemporal dementia. The trial planned to include many participants but was terminated ear…

This study tested whether Thealoz Total eye drops can reduce inflammation and improve symptoms in people with chronic dry eye disease. The drops contain hyaluronic acid for lubrication, plus trehalose and NAAGA to fight inflammation. The trial planned to enroll 30 adults with mod…

This study compared two different eye drops (Systane PRO and Refresh Optive Mega-3) to see which one works better for people with dry eye. Twenty adults with moderate dry eye symptoms were enrolled, but the study was stopped early. Participants filled out questionnaires about the…

This study tested whether a device called Nanodropper, which makes smaller eye drops, works as well as standard eye drops for lowering eye pressure in people with glaucoma or ocular hypertension. The study enrolled 16 adults who already used a common glaucoma drop. The goal was t…

This study tested a redesigned lacrimal stent, called the Slit Stent II, for people with severe tearing (epiphora) caused by a blocked tear duct. The stent has slits to allow tears to drain while it is still in place after DCR surgery. The trial planned to enroll 5 people to chec…

This study looked at whether a preservative-free eye gel called IRIDIUM® could help relieve dry eye symptoms in people with glaucoma or high eye pressure who were already using multiple eye drop medications. The goal was to see if the gel improved comfort and quality of life. The…

This study tested a device called CoolStat that is placed in the nose to cool down brain-injured patients with fever in the ICU. The goal was to see if it could safely and quickly bring their temperature to normal. Only 9 patients were enrolled before the study was stopped early,…

This study gave free eyeglasses to over 1,800 students aged 9-20 in Sierra Leone who had trouble seeing. Researchers wanted to find out if wearing glasses would improve their math and reading skills, and also help with anxiety and social well-being. The study was planned to last …

This study tested a cannabidiol (CBD) oral solution in children under 2 years old with tuberous sclerosis complex, Dravet syndrome, or Lennox-Gastaut syndrome who had uncontrolled seizures. The goal was to see if CBD is safe and can reduce seizures. However, the study was termina…

This study compared the TearCare system, a device that applies heat to the eyelids, to standard warm compresses and lid massage for people with dry eye disease. The goal was to see if TearCare is safe and better at improving tear stability and reducing eye discomfort. The study p…

This early-phase trial tested a new eye drop called rhPRG4 in 8 people with Sjögren's syndrome-related dry eye. Participants received either the active drop or a placebo, then switched after 90 minutes. The study measured eye dryness and safety over a single day. Because the tria…

This study aimed to see if certain eye tests could predict which people with dry eye disease would get relief from over-the-counter artificial tears. Researchers planned to enroll 66 adults with dry eye, but the study was terminated early with only 36 participants. Participants u…

This study tested a monthly solution called Tangible Boost that is meant to restore a special coating on contact lenses. The goal was to see if it could improve comfort and vision in people with severe dry eye from Stevens-Johnson syndrome, Sjogren's syndrome, or graft-versus-hos…

This study looks at whether people with Fabry, Gaucher, or Hunter disease are more likely to continue their IV treatment when it's given at home versus at a hospital. Researchers will review existing data from 222 patients in Mexico. No new treatments are given; the goal is to un…

This study follows people with geographic atrophy, a severe form of age-related macular degeneration that causes blind spots, to see how their vision changes over time. Researchers will measure visual function, such as reading speed and sensitivity to light, and track the growth …

This study checks on people who previously received gene editing for hemophilia B or mucopolysaccharidosis (MPS) I or II. No new treatment is given; instead, participants are monitored for up to 10 years to see if any new health problems or worsening of existing conditions appear…

This study looks at rare genetic disorders where the body can't make cholesterol properly, which can cause birth defects and learning problems. Researchers collect blood, urine, and tissue samples from affected people and their families to learn more about these conditions. The g…

This study is not testing a new treatment. Instead, it observes how 100 adults with a rare autoimmune disease called NMOSD are managed in everyday Russian clinics. Researchers will collect medical records and follow patients over time to understand their symptoms, treatments, and…

This study aimed to collect real-world data on Netherton Syndrome, a rare genetic skin disorder. Researchers planned to follow 4 participants over 52 weeks, measuring skin severity and other symptoms. The study was terminated early, so results are limited.

This study aimed to measure how eye volume changes (a sign of pressure fluctuation) before and after cataract surgery in patients with exfoliative glaucoma, a type of glaucoma common in Brittany. Only 6 adults were enrolled before the study was terminated. The goal was to see if …

This study looked at people with cone disorders, a type of inherited eye disease that affects color vision and sharp sight. Researchers analyzed genetic changes of unknown significance to see if they cause disease. The goal was to improve genetic diagnosis, not to test a treatmen…

This study looked at whether people with glaucoma would keep doing vision tests at home on an iPad every two weeks for six months. Eight patients took part. The goal was to see how often they tested and if the home test could catch worsening vision. Researchers also asked patient…

This study aimed to see if a new device called the EyeCheck monitor can measure eye pressure as accurately as standard tools in people with glaucoma or suspected glaucoma. Twenty-two participants used the device during a clinic visit. The study was stopped early, so results are l…

This study looked at whether special eye scans (OCT-angiography) can predict how well the remaining kidney will work one year after kidney removal for cancer. Researchers enrolled 30 adults scheduled for total nephrectomy. The goal was to see if retinal blood vessel patterns coul…

This study looked at 212 patients who woke up after a cardiac arrest outside the hospital. Researchers compared getting a heart angiogram right away versus waiting 12-24 hours. The goal was to see if immediate imaging reduces the risk of death, shock, or another cardiac arrest. T…

This study looked at whether peeling a thin membrane inside the eye (the internal limiting membrane) during surgery for a detached retina helps patients see better and prevents scar tissue. The trial was stopped early after enrolling only 21 people, so the results are not strong …

This study tested a new, non-invasive eye imaging method called hyperspectral imaging. It takes pictures of the back of the eye using many different colors of light to find details not visible with standard cameras. About 679 adults with healthy eyes or eye diseases like diabetic…

This study looked at whether measuring a protein called S100B in the blood right after a first seizure could help predict if someone will have more seizures within a year. The researchers planned to follow 37 adults for one year, but the study was stopped early. The goal was to f…

This study aimed to validate a scoring system that predicts whether patients will have good vision one year after combined cataract and epiretinal membrane surgery. The score uses factors like age, symptom duration, and initial vision. Researchers planned to enroll 164 adults age…

This study followed 7 people with Mucolipidosis Type IV (MLIV) to learn how the disease naturally progresses. Researchers used tests like neuropsychological exams, blood and urine tests, and brain MRIs to find better ways to measure the disease. The goal was to improve future cli…

This study looked at over 100 people with inherited retinal dystrophies, a group of rare eye diseases that can cause vision loss. Researchers collected genetic and eye exam data to find links between specific gene mutations and symptoms. The goal was to better understand these di…

This study compared two methods of removing the clear outer layer of the eye (the epithelium) during laser vision correction for nearsightedness and astigmatism. One method uses a laser, the other uses alcohol and manual scraping. The goal was to see which approach leads to less …

This study aimed to find out how many people with Sjögren's dry eye have reduced feeling in their cornea (the clear front part of the eye). Researchers measured corneal sensitivity, tear production, and pain in 103 adults with confirmed Sjögren's syndrome and dry eye. The study w…

This pilot study aimed to see if antiseizure drugs like phenobarbital and levetiracetam change brain connectivity patterns in people with severe acute brain injury and reduced consciousness. Only 5 participants were enrolled before the study was terminated. Researchers used resti…

This study looked at how well the COVID-19 vaccine triggers an immune response in people with autoimmune or inflammatory diseases who take immunosuppressants or biologics. Researchers took blood samples before vaccination and at several points after to measure antibodies and T-ce…

This study aimed to collect eye scan data from people with and without glaucoma to develop a new screening tool called the Topcon Health Score. The researchers planned to use a special camera to measure the thickness of nerve layers in the eye. However, the study was terminated e…

This study follows up on children with Krabbe disease who received a one-time gene therapy infusion (FBX-101) in earlier trials. Researchers will monitor safety and measure motor skills over time. Only 2 participants are enrolled, so results are very limited.