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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.

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  1. Early Access Program With Arimoclomol in US Patients With NPC

    Rochester, MN

  2. A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

    Rochester, MN

  3. Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C

    Rochester, MN

  4. A Study to Establish a Rare Diseases Registry for Niemann-Pick Disease Types A, B and C

    Rochester, MN


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