Usher syndrome type 1B
MONDO:0700087Usher syndrome in which the cause of the disease is a mutation in the MYO7A gene
Also known as: Usher syndrome, type 1B
3 clinical trials for this condition and its sub-types.
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Gene therapy for rare Blindness-Deafness syndrome passes early safety check
Disease control OngoingThis phase 2 study follows 9 adults with Usher syndrome type 1B who previously received a single injection of the gene therapy SAR421869 under their retina. Researchers are monitoring long-term safety and whether the treatment can slow retinal degeneration. The trial is active bu…
Phase: PHASE2 • Sponsor: Sanofi • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC