All results
26 results
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Clinical Trials
Adult patients with Gaucher Disease Type 1 who have previously received SRT (substrate replacement therapy) and are now receiving ERT (enzyme replacement…
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Clinical Trials
Individuals diagnosed with COVID-19 are being asked to take part in a research study of an investigational drug called Asapiprant (BGE 175). This drug is being…
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Clinical Trials
A Natural History and Outcome Measure Discovery Study of PRPF31 Mutation–Associated Retinitis Pigmentosa
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Clinical Trials
This multi-center, randomized, double-blind, placebo-controlled, dose-range-finding clinical trial will assess the efficacy, safety, and tolerability of…
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Clinical Trials
This is a Phase 3, double-blind, randomized, placebo-controlled trial of Reldesemtiv in patients aged 18 to 80 with ALS. A drug named "Reldesemtiv" is being…
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Clinical Trials
This research study is being done to learn more about how well an investigational study drug, called otilimab, works and how safe it is when taken for a longer…
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Clinical Trials
This randomized trial will evaluate the effect of fenofibrate compared with placebo for prevention of diabetic retinopathy (DR) worsening or center-involved…
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Clinical Trials
The purpose of this research study is to assess the safety, tolerability (primary objective), efficacy, and target engagement (secondary objective) of…
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Clinical Trials
Ashvattha Therapeutics, Inc. has begun a clinical study of an investigational drug (also known as the "study drug") called OP-101 as a possible treatment for…
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Clinical Trials
Your child was born, or is expected to be born, prematurely. Premature infants can get a serious inflammatory condition, called necrotizing enterocolitis…