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During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

Search Results 1-2 of 2 for Tay-Sachs disease

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  • Study status: Open/Status Unknown
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  1. A Study to Evaluate the Effects of N-Acetyl-L-Leucine for GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease)

    Rochester, MN

  2. A Study of Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidosis, or Gaucher Disease Type 2 in Pediatric Patients

    Rochester, MN

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