ORBIT Study - UX143Summary: A clinical trial for children, adolescents, and young adults living with osteogenesis imperfecta (OI)
sEARCH RESULTS
AT1001-030
AT1001-030Summary: followME is a disease registry that will track the health of people living with Fabry disease over time. The goal is to evaluate how different types of disease management affect people living with Fabry.
SYNB1934-CP-003
SYNB1934-CP-003Summary: The goal of this new study is to evaluate an investigational drug called SYNB1934.
FACTS
FACTSSummary: Phase I gene therapy for Fabry Disease
REN001
REN001Summary: A double-blind, placebo-controlled, study to evaluate the efficacy and safety of 24 weeks treatment with REN001 in patients with primary mitochondrial myopathy (PMM).
PTC923-MD-004-PKU
PTC923-MD-004-PKUSummary: A phase III study of PTC 923 in subjects with Phenylketonuria
AVRO-RD-02-LTF01
AVRO-RD-02-LTF01 A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02 Summary: A follow-up study of subjects with Type I Gaucher Disease who previously received AVR-RD-02 gene therapy
GRIT STUDY
G.R.I.T. STudyGetting global Rare disease Insights through Technology Summary: The G.R.I.T. Study is the first global app-based registered clinical trial for patients with metabolic disorders. It uses a mobile health journal, Zamplo, to record patient-reported...
MIGALASTAT SWITCH
MIGALASTAT SWITCHSummary: Evaluation of Biomarkers for patients receiving migalastat after switching from ERT.