303. アッシャー症候群 Usher syndrome Clinical trials / Disease details


臨床試験数 : 10 薬物数 : 11 - (DrugBank : 0) / 標的遺伝子数 : 0 - 標的パスウェイ数 : 0

  
1 trial found
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1NCT05085964
(ClinicalTrials.gov)
September 16, 20217/10/2021An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis PigmentosaAn Open-Label Extension Study to Evaluate the Safety and Tolerability of QR 421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Helia)Retinitis Pigmentosa;Usher Syndrome Type 2Drug: RNA antisense oligonucleotide for intravitreal injectionProQR TherapeuticsNULLTerminated12 YearsN/AAll21Phase 2United States;Canada;France