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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.

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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).

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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

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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...

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