4 results

• Randomized

  Trial design where participants are randomly assigned to treatment or control groups, ensuring unbiased comparison of outcomes.

  Reference:

  EU Clinical Trials Register. Glossary [Internet]. [cited 2025 Nov]. Available from: https://www.clinicaltrialsregister.eu/doc/EU_Clinical_Trials_Register_Glossary.pdf

• Rare disease

  A disease is generally defined as rare when it affects fewer than 1 in 2,000 individuals in the general population (in the EU, fewer than 5 in 10,000 people; in the US, fewer than 200,000 people in the US).

  Reference:

  Haendel M, Vasilevsky N, Unni D, et al. How many rare diseases are there? Nat Rev Drug Discov. 2020;19(2):77-78. doi:10.1038/d41573-019-00180-y

• Real-world studies (RWS)

  Research process that uses health-related data collected outside the strict controls of clinical trials.

  Reference:

  Costa V, Custodio MG, Gefen E, Fregni F. The relevance of real-world evidence in research, clinical, and regulatory decision making. Front Public Health. 2025;13:1512429. doi:10.3389/fpubh.2025.1512429

• Risk factor

  Any attribute, characteristic, or exposure that increases an individual's vulnerability to developing a disorder. In PSP, environmental, genetic and epigenetic risk factors, alongside age-related changes, are thought to interact and contribute to the disease; however, the exact underlying cause of this tauopathy is unknown.

  Reference:

  ScienceDirect Topics. Risk factor [Internet]. [cited 2025 Nov]. Available from: https://www.sciencedirect.com/topics/agricultural-and-biological-sciences/risk-factor | Park HK, Ilango SD, Litvan I. Environmental risk factors for progressive supranuclear palsy. J Mov Disord. 2021;14(2):103-113. doi:10.14802/jmd.20173