Search for information about a product,
therapeutic area or event

Search

Media, investors, advocacy organizations and others, please contact us here.

Posters

A Quantitative Systems Pharmacology (QSP) Model for Classical Homocystinuria Predicting Efficacy of Treatment

Learn More
Posters HCU

Development of a Patient Identification Algorithm to Estimate Prevalence of Classical Homocystinuria (HCU) in the United States (US)

Learn More
Posters HCU

Insights From the First Genetic Evaluation of a Longitudinal Natural History Study in Classical Homocystinuria (HCU)

Learn More
Posters HCU

Population-based Incidence Estimates of Classical Homocystinuria Using the Genome Aggregation Database (gnomAD)

Learn More
Posters

Skeletal Fragility In A Natural History Study Of Homocystinuria Due To Cystathionine Beta-synthase Deficiency Patients

Learn More
Posters

Comorbid Diagnoses, Medications, and Healthcare Provider Types among Patients Diagnosed with Classical Homocystinuria

Learn More
Posters

Long-Term Efficacy and Safety of Sparsentan in Young Patients With FSGS: 240-Week Analysis of the DUET Open-Label Extension (OLE)

Learn More
Posters

Alport Syndrome Natural History from the Rare Disease Registry: Associations with gene, variant type, and sex

Learn More
Posters IgAN

Estimating delay in time to kidney failure ordeath for treatment effects on proteinuria inIgA nephropathy

Learn More
Posters IgAN

Matching-adjusted indirect comparison of sparsentan vs. delayed-release formulationbudesonide for proteinuria reduction in adults with IgA nephropathy

Learn More
Posters FSGS

Understanding the Impact of Immunoglobulin ANephropathy (IgAN) and Focal Segmental Glomerulosclerosis (FSGS) on Patients and Care-Partners Study(HONUS): Updated Results for IgAN in the United States (US)

Learn More
Posters CTX

Prevalence of Cerebrotendinous Xanthomatosis (CTX) Among Patients Diagnosed With Juvenile-Onset Idiopathic Bilateral Cataracts

Learn More
Posters

Genetic Testing Identified Causative Variants in Nearly 20% of Early-Onset Bilateral Cataracts Cases

Learn More