Neurology and Amyloid-Related Disorders
Cerebral amyloid angiopathy, Alzheimer’s disease, and early-onset Alzheimer’s disease are central areas of interest, alongside Huntington’s disease. The clinical portfolio also includes studies in amyloid-related brain vascular disease and neurodegenerative conditions linked to abnormal protein accumulation.
- Cerebral amyloid angiopathy
- Alzheimer’s disease
- Early-onset Alzheimer’s disease
- Huntington’s disease
Research activity in this area reflects a focus on neurological impairment, cognitive decline, and vascular complications associated with amyloid pathology.
Transthyretin Amyloidosis
Hereditary transthyretin amyloidosis, hereditary transthyretin-mediated amyloidosis with polyneuropathy, and transthyretin amyloidosis with cardiomyopathy form a major therapeutic domain. The sponsor also studies systemic manifestations of transthyretin-driven disease across neurologic and cardiac involvement.
- Hereditary transthyretin amyloidosis
- Hereditary transthyretin-mediated amyloidosis with polyneuropathy
- Transthyretin amyloidosis with cardiomyopathy
This area includes both neuropathy and cardiomyopathy endpoints, highlighting interest in multisystem amyloid disorders.
Cardiovascular Medicine
Hypertension, established cardiovascular disease, and high cardiovascular risk are prominent areas of study, with additional attention to outcomes such as myocardial infarction, stroke, and heart failure. Cardiovascular investigations also intersect with amyloidosis-related cardiac disease.
- Hypertension
- Established cardiovascular disease
- High cardiovascular risk
- Heart failure
The sponsor’s cardiovascular research spans risk reduction and organ-specific complications, including cardiac involvement in amyloid disorders.
Renal and Metabolic Disorders
Primary hyperoxaluria type 1 is a key renal-metabolic focus, with studies addressing oxalate burden in patients with and without dialysis. The portfolio also extends to type 2 diabetes mellitus, reflecting interest in metabolic disease management.
- Primary hyperoxaluria type 1
- Type 2 diabetes mellitus
These programs connect kidney disease, metabolic control, and systemic complications that require long-term disease management.
Vascular and Rare Genetic Conditions
Hereditary hemorrhagic telangiectasia and hepatocellular carcinoma appear among the sponsor’s additional research areas, broadening the portfolio into vascular malformation and oncology. These studies sit alongside inherited and rare-disease programs across the company’s clinical activity.
- Hereditary hemorrhagic telangiectasia
- Advanced or metastatic hepatocellular carcinoma
Interest in these conditions adds coverage of vascular fragility and liver cancer within the wider rare-disease landscape.
