Primary Hyperoxaluria
Clinical research activity is centered on primary hyperoxaluria, with a focus on PH1 and the kidney-related complications that accompany the disorder.
- Oxalate reduction
- Renal impairment
- Dialysis-associated care
Interest extends to patients with severe kidney dysfunction, including those receiving hemodialysis or peritoneal dialysis.
Renal Function Preservation
The sponsor’s studies examine the impact of treatment on estimated glomerular filtration rate and other markers of renal function in people living with PH1.
- eGFR
- Chronic kidney disease
- Kidney injury
These clinical interests reflect a strong emphasis on protecting remaining kidney performance in a rare metabolic nephrology setting.
Rare Metabolic Disease
The research portfolio is concentrated on a single rare inherited condition, highlighting therapeutic work in inborn errors of metabolism with renal manifestations.
- Inherited metabolic disorders
- Oxalate overproduction
- Systemic disease burden
Clinical interest is directed toward disease mechanisms that drive excess oxalate accumulation and downstream kidney complications.
Long-Term Safety and Tolerability
Trials also address the long-term use of DCR-PHXC, with attention to ongoing safety, tolerability, and sustained clinical effect in primary hyperoxaluria.
- Long-term treatment exposure
- Safety monitoring
- Therapeutic durability
This area includes patients with advanced renal impairment and those requiring dialysis support.
