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

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).

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

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.

Rapid eye movements that repositions the eyes to a new location.

The overall assessment of a drug’s adverse effects, tolerability, and risk characteristics.

The number of subjects involved in a study. A larger sample size provides stronger evidence, as it is associated with greater statistical power.

Rapid eye movements that repositions the eyes to a new location.

A natural process in cells that edits genes by cutting out unnecessary parts and joining the useful sections together to make a complete instruction for building a protein.

Care given to patients that includes symptomatic treatment but also focuses on improving overall quality of life. In PSP, supportive care can involve physical, occupational and speech therapy and nutritional and psychological support to help patients manage the disease and maintain their independence for as long as possible.

Impaired voluntary eye movements, especially vertical gaze, more than in the horizontal plane, affecting both up- and downgaze.

Interventions aimed at alleviating symptoms of a disease rather than addressing the underlying cause.

Refers to the proportion of brain OGA enzyme bound by FNP-223, measured via PET imaging.

Tau protein is a protein in the cytoskeleton of neurons that is key to stabilizing microtubules, which act as axonal transport pathways and form part of the neuronal structure. In tauopathies, tau protein loses its normal function, and forms NFTs, which disrupt cellular transport and lead to neurodegeneration and neuronal death. Loss of normal tau function and abnormal aggregation are central to PSP pathophysiology; and the modulation of tau O-GlcNAcylation is a therapeutic strategy under investigation.

A group of neurodegenerative disorders characterized by tau deposits in the brain, with symptoms of dementia and parkinsonism.

The remote monitoring and recording of physiological parameters, such as heart rate or ECG, to assess a patient’s long-term functions in real time.

The elapsed time after drug administration at which the highest concentration of the drug in plasma (Cmax) is observed.

The degree to which adverse effects of a treatment can be tolerated by patients without experiencing significant adverse events.

A radioactive compound used in PET imaging to bind selectively a protein. For example, the [18F]-IMA601 binds to the O-GlcNAcase (OGA) enzyme, allowing visualization and measurement of OGA distribution and target engagement in the brain.

An adverse event that appears or worsens during a clinical trial. It helps identify events that are temporally associated with the drug.

A distinct type of astrocytic pathology characterized by the presence of dense, argyrophilic (silver stain-positive) phosphorylated tau-immunopositive inclusions within the proximal processes of astrocytes. Tufted astrocytes are a histopathological hallmark of PSP.

Aspects of care or disease management that currently have no adequate solutions (such as misdiagnosis or lack of curative therapies in the case of PSP).

A magnetic resonance imaging technique that quantifies the volume of specific brain structures to assess structural changes over time.