Alzheimer’s Disease and Neurodegeneration
Research activity includes early Alzheimer’s disease and other disorders affecting cognitive decline and brain function, with interest in central nervous system biomarkers and neuroprotective approaches.
- Early Alzheimer’s disease
- Central nervous system activity
- Neurodegenerative disease
The portfolio reflects a focus on therapies intended to address disease processes within the brain and support measurable neurological outcomes.
Frontotemporal Dementia
Clinical research includes frontotemporal dementia (FTD), with attention to genetic forms such as FTD-GRN and associated changes in behavior, cognition, and neurological function.
- Frontotemporal dementia (FTD)
- FTD-GRN
- Behavioral and cognitive impairment
These studies are centered on therapies for inherited and progressive forms of dementia that affect executive function and daily behavior.
Mucopolysaccharidosis Type II and Hunter Syndrome
The sponsor is active in mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, including both pediatric and young adult populations and both neuronopathic and non-neuronopathic disease forms.
- Mucopolysaccharidosis type II (MPS II)
- Hunter syndrome
- Pediatric lysosomal storage disorders
Research in this area emphasizes treatment effects on cognitive function, adaptive behavior, and long-term disease management.
Parkinson’s Disease and LRRK2-Related Disorders
Trials also address Parkinson’s disease, including genetically defined LRRK2-associated Parkinson’s disease, with interest in motor and non-motor disease manifestations.
- Parkinson’s disease
- LRRK2-associated Parkinson’s disease
- Neurological movement disorders
This area reflects involvement in therapies for inherited forms of parkinsonism and related neurodegenerative movement disorders.
Rare Disease and Neurology
The broader research landscape spans rare neurological diseases with strong emphasis on central nervous system involvement, genetic disease mechanisms, and disorders affecting both children and adults.
- Rare neurological disease
- Genetic disorders
- Central nervous system therapeutics
Current activity covers conditions where neurological function, behavior, and disease-specific biomarkers are central to therapeutic development.
