Your role in research starts here

ORBIT-CIDP

Enrolling

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

VISTA

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

Enrolling

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

PLEDGE

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.

PERFORM Patient Registry

Enrolling

The PERFORM Patient Registry is an observational research effort to gather insights into the day-to-day lives of individuals taking TARPEYO™.

CAHtalog

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.

CureDuchenne Link

Enrolling

Change the future of DMD/BMD just by sharing your or your child's health history with the brightest minds in medicine.

Breast Cancer Registry

Enrolling

The Breast Cancer Registry study is an observational research effort to gather insights into the day-to-day lives of individuals with breast cancer.

SarChart

Enrolling

SarChart is a patient-powered observational study designed to create a holistic picture of the LGMD journey – including quality of life and care.

ASMD Accelerate

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.