Your role in research starts here

Compass Study

Enrolling

This observational study is designed to better understand the impact of MMA and PA on families. By collecting medical records and brief survey responses, researchers can understand how the condition is managed, how symptoms evolve, and where support is most needed.

Navigate-CIDP Study

Enrolling

The NAVIGATE-CIDP Study looks at how CIDP affects a person’s daily life, based on symptoms and quality-of-life surveys. It also tracks treatment patterns—like why people switch medications—and builds a foundation for future research.

Beacon SLE Study

Enrolling

The BEACON-SLE Study is a real-world non-interventional research study that looks at the experiences of people with systemic lupus erythematosus (SLE) and how they respond to treatment with Benlysta® (belimumab) in real life.

OBSERVE-NMO Study

Enrolling

The OBSERVE-NMO Study is a real-world observational research study that looks at the experiences of people with NMOSD who take UPLIZNA® (inebilizumab-cdon) to improve future care.

ARIES Study

Enrolling

The ARIES (Assessing Real-World Experiences on SKYCLARYS®) Study will help researchers better understand how omaveloxolone works in people living with Friedreich’s ataxia.

Tepli-QUEST Study

Enrolling

The Tepli-QUEST Study is a real-world observational research study that looks at the experiences of people with Stage 2 type 1 diabetes (T1D) who have received teplizumab compared to those who have not.

ORBIT-CIDP Study

Enrolling

The ORBIT-CIDP study collects real-world data to better understand CIDP, including condition progression, treatment effectiveness, and patient experiences.

VISTA Study

Enrolling

VISTA is an observational study designed to create a holistic picture of the achondroplasia journey for children – including quality of life and care.

OPUS-W Study

Enrolling

The OPUS-W registry combines anonymized data from patients with ATTR taking WAINUA™ to gain a clearer picture of their daily life.

PLEDGE Study

Enrolling

The PLEDGE Study is an observational research effort to gather insight into the day-to-day lives of individuals who recently initiated danicopan to manage PNH.

CAHtalog Study

Enrolling

CAHtalog’s mission is to advance CAH clinical research and in turn improve the quality of life for those living with classic CAH.

Long-Term Follow-Up Study

Enrolling

This study will follow your health over time to better understand the long-term effects and outcomes after receiving gene-modified T-cell therapy.

ASMD Accelerate Study

Enrolling

This study is hoping to understand how ASMD progresses over time, as well as how the disease is actually managed and treated in the real-world.