USHER SYNDROME AND RETINITIS PIGMENTOSA (USH1C and RP)
McGill and MUHC team up with LSU Health New Orleans to advance Usher Syndrome antisense therapy research
USHER SYNDROME AND RETINITIS PIGMENTOSA (USH2A and RP)
Antisense genetic treatment: A First in Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) and Usher syndrome due to Mutations in Exon 13 of the USH2A Gene
https://publications.mcgill.ca
AGE RELATED MACULAR DEGENERATION (AMD)
Anti-complement drug treatment in DRY AMD: Genetic studies on exudative Age-related macular degeneration (AMD) patients receiving intra-vitreal Ranibizumab injections.
LEBER CONGENITAL AMAUROSIS (LCA)
A new antisense genetic treatment for CEP290 patients: 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
https://www.proqr.com/sepofarsen-for-lca10/
RETINITIS PIGMENTOSA (RP)
New stem cell treatment for Retinitis pigmentosa, showing acuity improvements and a natural history of the progression of X-Linked Retinitis Pigmentosa.
CHOROIDEREMIA (CHM)
A Randomised, Open-Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial of Retinal Gene Therapy for Choroideremia Using An Adeno-Associated Viral (AAV2) Encoding Rab Escort Protein 1 (REP1)
PEROXISOME DISORDERS
Getting Ready for Vision Therapy in Peroxisome Biogenesis Disorders