Rare Disease

(264) A Non-Interventional Study Using Multiple Data Sources to Evaluate Potential Sight Threatening Events and Severe Visual Loss Among Patients Exposed to Crizotinib

(263) Clinical Evaluation of Code-Based Algorithms to Identify Pulmonary Arterial Hypertension Patients in Coded Healthcare Databases

(AG) A Population-Based Study on The Prevalence of Diagnosed Primary Immune Thrombocytopenia in the United States, 2016-2021

(AH) Algorithm to Identify Patients with Hypereosinophilic Syndrome (HES) in Electronic Health Record Databases

(AK) An Innovative Way to Generate Blinded Data in Single Arm Clinical Trials

(AF) Development and Testing of Diagnostic Algorithms for Identifying Subjects with Acromegaly in Administrative Claims Databases

(AM) Natural History of Wilson's Disease in the United States Using Optum® Electronic Health Records

(AI) Risk of Hospitalization for Vaso-Occlusive Episode in Patients with Sickle-Cell Disease After Out-Patient Exposure to Systemic Corticosteroids

(AJ) The Use of United States Administrative Claims Data to Study Patients with Friedreich Ataxia: The Impact of Specific and Non-Specific Coding Periods

(AL) Use of Provider Notes in Novel Identification of Cancer at The Gastroesophageal Junction

(254) Epidemiology of Palmoplantar Pustulosis in Urban China and a Systematic Review

(262) Frequency of Sleep Disorders and Insomnia in Patients with Wilson Disease Identified in Electronic Health Records

(259) Marketing Authorisation Pathways and Clinical Evidence Supporting Approval of Orphan Medicinal Products in EU Between January 2015 and October 2021

(261) Outcome Measures Relevant for Assessing Health Related Quality of Life and Utility in Friedreich Ataxia

(257) Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Literature Review

(258) Prevalence of Postural Orthostatic Tachycardia Syndrome (POTS) in the US

(252) Sarcoidosis in Patients After Solid Organ Transplantation Treated with mTOR Inhibitors Versus Calcineurin Inhibitors

(255) Survival Benefit of Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria

(253) The Burden of AADC Deficiency on Patients, Caregivers, and Healthcare Systems: A Systematic Literature Review

(260) The Development of a Conceptual Model to Describe the Health-Related Quality of Life Impacts of Living with Friedreich Ataxia

(256) Treatment Patterns for Depression and Anxiety Among Patients with Progressive Supranuclear Palsy (PSP)