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ORBIT-CIDP
Enrolling
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

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

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Medical Record Review
Smart Insoles (Optional)
Surveys
Virtual Assessments
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VISTA
Enrolling
Achondroplasia

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

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Medical Record Review
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OPUS-W
Enrolling
Transthyretin-Mediated Amyloidosis (ATTR)
WAINUA™

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

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Medical Record Review
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PLEDGE
Enrolling
Paroxysmal Nocturnal Hemoglobinuria (PNH)
danicopan

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.

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Medical Record Review
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IgA Nephropathy (Berger's Disease)
TARPEYO™

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

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Medical Record Review
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CAHtalog
Enrolling
Congenital Adrenal Hyperplasia (CAH)

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

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Medical Record Review
Surveys
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Duchenne & Becker Muscular Dystrophy

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

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Medical Record Review
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Breast Cancer (Stages I-III)

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

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Medical Record Review
Surveys
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SarChart
Enrolling
Limb-Girdle Muscular Dystrophy

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

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Medical Record Review
Surveys
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Acid Sphingomyelinase Deficiency

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.

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Medical Record Review
Surveys
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