Rate of Progression in USH2A Related Retinal Degeneration | Tübingen |
Rate of Progression in EYS Related Retinal Degeneration | Tübingen |
Rate of Progression of PCDH15-Related Retinal Degeneration in Usher | Tübingen |
Natural history of the progression of X-linked retinitis pigmentosa. | Tübingen |
Bringing Gene Supplementation Therapy for Inherited PDE6A- and CNGA3-associated Retinopathies into Clinical Practice: A joint Tübingen-München Project Acronym (www.rd-cure.de) | Tübingen |
Belite Bio = DRAGON = Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects | Tübingen |
A Phase 1/2, Open-Label, Dose Escalation and Dose Expansion Study to Evaluate the Safety and Tolerability of ATSN-201 Gene Therapy in Male Subjects with RS1-Associated X-linked Retinoschisis | Tübingen |
Analyse der klinischen Untersuchungen von Patienten mit Netzhautdystrophien nach Behandlung mit Voretigene neparvovec (LuxturnaTM) | Tübingen |
Klinische Charakterisierung BEST Erkrankung | Tübingen |
CNGB1 Retinitis Pigmentosa and Allied Disorders | Tübingen |
Klinische Charakterisierung CNGA1 Retinitis pigmentosa | Tübingen |
Clinical and Socio-demographic Characteristics of Patients with X-linked- Retinitis Pigmentosa (XLRP) and Impact of the Disease | Tübingen |
Study to Determine Presence of Genetically Confirmed CNGA3- and CNGB3-Associated Achromatopsia Patients at Investigational Sites for Potential Participation in Future Clinical Research Studies | Tübingen |
Retrospective Non-Interventional Study to Evaluate the Patient Benefit of Transcorneal Electrostimulation (TES) | Tübingen |
Observational Study for Patients Treated with voretigene Nepravovec | Tübingen |
A double-masked, randomized, controlled, multiple-dose study to evaluate the efficacy, safety, tolerability and systemic exposure of QR-110 in Subjects with Leber’s Congenital Amaurosis (LCA) due to c.2991+1655A>G mutation (p.Cys998X) in the CEP290 gene (Illuminate). | Tübingen |
Safety and efficacy of a bilateral single subretinal injection of rAAV.hCNGA3 in adult and minor patients with CNGA3-linked achromatopsia investigated in a randomized, wait list controlled, observer-masked trial | Tübingen |
Multi-national, multi-centre, double-masked, placebo-controlled proof of concept trial to evaluate the safety and efficacy of oral soraprazan in Stargardt disease | Tübingen |
A Phase 2, randomized, concurrent-controlled study of AAV8-hCARp.hCNGB3 gene therapy for the treatment of achromatopsia associated with variants in the CNGB3 gene | Tübingen |
An open-label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects of Age 3 to <8 Years with Leber Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene | Tübingen |
A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Early to Moderate Vision Loss | Tübingen |
A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Advanced Vision Loss | Tübingen |
Evaluation der Wirksamkeit einer transkornealen Elektrostimulation bei Patienten mit Retinitis Pigmentosa – eine multizentrische, prospektive, randomisierte, kontrollierte und doppel-blinde Studie im Auftrag des Gemeinsamen Bundesausschusses (G-BA) | Tübingen |
PDE6A gene therapy for retinitis pigmentosa | Tübingen |
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) | Gießen |
An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene. (BRIGHTEN) | Gießen |
Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene | Gießen |
Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene | Gießen |
A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late-onset GM2… | Gießen |
A Phase 3, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis | Gießen |
Percieve – Nicht-interventionelle Studie (NIS) zur Beobachtung von Patienten mit RPE65-assoziierter Netzhautdegeneration, die mit dem Medikament LTW888 behandelt wurden. Ziel ist es, die Unbedenklichkeit und den Langzeitverlauf der Behandlung zu untersuchen. | Gießen |