Listado de ensayos


Auditory Neural Function in Implanted Patients With Usher Syndrome

Estado: | Categoría: | Resultado:

Patología

Usher Syndrome Cochlear Implantation

Tratamiento

Other: Experimental manipulation of stimulation parameters

Localizaciones

Boston children's Hospital, Boston, Massachusetts, United States Gina Hounam, Columbus, Ohio, United States The Ohio State University, Columbus, Ohio, United States Vanderbilt University Medical Center, Nashville, Tennessee, United States
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Classification, Functional Stratification and Biomarkers in Ciliopathy (CILLICORIRCM)

Estado: | Categoría: | Resultado:

Patología

Ciliopathies Nephronophthisis Senior-Loken Syndrome Joubert Syndrome Jeune Syndrome Bardet-Biedl Syndrome

Tratamiento

Other: Blood sample Other: Urine sample

Localizaciones

H pital Necker-Enfants Malades, Paris, France
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A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B

Estado: | Categoría: | Resultado:

Patología

Usher's Syndrome

Tratamiento

Drug: Blood draw for the laboratory assessment

Localizaciones

Investigational Site Number 840001, Portland, Oregon, United States Investigational Site Number 250001, Paris, France
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Stem Cell Ophthalmology Treatment Study

Estado: | Categoría: | Resultado:

Patología

Retinal Disease Macular Degeneration Hereditary Retinal Dystrophy Optic Nerve Disease Glaucoma

Tratamiento

Procedure: RB (Retrobulbar) Procedure: ST (Subtenon) Procedure: IV (Intravenous) Procedure: IVIT (Intravitreal) Procedure: IO (Intraocular)

Localizaciones

MD Stem Cells, Westport, Connecticut, United States
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Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

Estado: | Categoría: | Resultado:

Patología

Inherited Retinal Dystrophy Due to RPE65 Mutations Leber Congenital Amaurosis

Tratamiento

Biological: AAV2-hRPE65v2,voretigene neparvovec-rzyl

Localizaciones

University of Iowa, Iowa City, Iowa, United States Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
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Clinical and Genetic Examination of Usher Syndrome Patients’ Cohort in Europe

Estado: | Categoría: | Resultado:

Patología

Usher Syndrome

Localizaciones

CHU, Laboratoire de g n tique mol culaire, INSERM, Montpellier, France CIC of CHNO des Quinze-vingts, Paris, France Johannes Gutenberg University of Mainz, Institute of Zoology, Dept. Cell and Matrix Biology Mainz, Mainz, Germany Radboud University Nijmegen Medical Centre, Dept. Otorhinolaryngology, Nijmegen, Netherlands AIBILI, 4C - Coimbra Coordinating Centre for Clinical Research, Coimbra, Portugal IBILI- Faculty of Medicine - University of Coimbra, Center for Hereditary Diseases and Visual Neurosciences Laboratory, Coimbra, Portugal
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A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

Estado: | Categoría: | Resultado:

Patología

Confirmed Biallelic RPE65 Mutation- associated Retinal Dystrophy

Tratamiento

Biological: AAV2-hRPE65v2,voretigene neparvovec-rzyl

Localizaciones

Children's Hospital of Los Angeles, Los Angeles, California, United States Bascom Palmer Eye Institute, Miami, Florida, United States University of Iowa Hospitals & Clinics, Iowa City, Iowa, United States Massachusetts Eye and Ear Institute, Boston, Massachusetts, United States Kellogg Eye Center, Ann Arbor, Michigan, United States Cincinnati Eye Institute, Cincinnati, Ohio, United States Casey Eye Institute, Portland, Oregon, United States Children's Hospital of Philadelphia (CHOP), Philadelphia, Pennsylvania, United States Scheie Eye Institute, Philadelphia, Pennsylvania, United States Cullen Eye Institute, Houston, Texas, United States
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Rate of Progression in USH2A-related Retinal Degeneration

Estado: | Categoría: | Resultado:

Patología

Usher Syndrome, Type 2A RETINOSIS PIGMENTARIA 39

Localizaciones

University of California, San Francisco, San Francisco, California, United States Vitreo-Retinal Associates, Gainesville, Florida, United States Wilmer Eye Institute at Johns Hopkins, Baltimore, Maryland, United States Massachusetts Eye and Ear, Boston, Massachusetts, United States Kellogg Eye Center, University of Michigan, Ann Arbor, Michigan, United States Duke University Eye Center, Durham, North Carolina, United States OHSU Casey Eye Institute, Portland, Oregon, United States Retina Foundation of the Southwest, Dallas, Texas, United States Baylor Eye Physicians and Surgeons, Houston, Texas, United States Moran Eye Center, University of Utah, Salt Lake City, Utah, United States and 6 more
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