Disease Space Cardiovascular Metabolic Lifestyle Disease Rare Disease
Disease Space Cardiovascular Metabolic Lifestyle Disease Rare Disease/ Pediatrics Model Market (Icons) Patient Impact & Market Size Important Information Atherosclerosis Devices/Drugs/Imaging Major cause of heart disease which causes 1 in 4 deaths in the US (610, 000/year) Links to publications (3 publications) FDA Enforcement Discretion Granted Dilated Cardiomyopathy (DCM) Devices/Drugs/Advanced Therapy/Biomarkers 1 in 2500 affected by DCM 30% of all DCM cases have a genetic cause, often inherited as an autosomal dominant trait Links to publications (1 publication in press) FDA Enforcement Discretion Granted Developed in partnership with Mayo Clinic Atrial Fibrillation (Afib) Devices/Drugs/Biomarkers 2. 7 to 6. 1 million people in US have Afib 158, 000 Americans die of Afib each year 2 different genotypes available Hypertension Devices/Drugs/Biomarkers Affects nearly half of all adults in the US Only 24% patients have their blood pressure controlled by medication Primary cause of death for 472, 000 people in 2017 in US Costs the US $131 billion each year 3 different genotypes available Nonalcoholic Steatohepatitis (NASH) Drugs/Advanced Therapy/Imaging/Biomarkers Afffects 5% of Americans Can lead to liver failure, liver cancer and death Funding supported by NIH Developed in partnership with Indiana University Type 1 Diabetes (T 1 D) Devices/Drugs/Advanced Therapy/Biomarkers 1. 6 million Americans have T 1 D Costs the US$16 billion annually Funding supported by NIH Developed in partnership with Indiana University Neurofibromatosis Type 1 (NF 1) Drugs/Advanced Therapy/Imaging/Biomarkers 1 in 3, 0000 children born have NF 1 Patients are predisposed to tumor development Links to publications (1 publication, 1 pending) FDA Enforcement Discretion Granted Developed in partnership with the Children’s Tumor Foundation and the Gilbert Family Foundation Neurofibromatosis Type 2 (NF 2) Drugs/Advanced Therapy/Imaging/Biomarkers 1 in 25, 000 children born have NF 2 Patients are predisposed to tumor development Funding supported by NIH-NINDS Developed in partnership with UCLA and the Children’s Tumor Foundation Microvillus Inclusion Disease (MVID) Drugs/Advanced Therapy/Biomarkers Very rare disease (less than 100 patients) Funding supported by NIH Osteogenesis Imperfecta (OI) Devices/Drugs/Advanced Therapy/Imaging/Biomarkers 1 in 15, 000 people have OI Leads to brittle bones Polycystic Kidney Disease (ARPKD & ADPKD) Drugs/Advanced Therapy 600, 000 patients in US Causes 5% of all kidney failure Funding supported by NIH Developed in partnership with Mayo Clinic Phenylketonuria (PKU) Drugs/Advanced Therapy 1 in 10, 000 babies born are diagnosed with PKU Link to publication (1 in press) Developed in partnership with Children’s Minnesota Funded supported by NIH Affects 100, 000 Americans Funding supported by NIH Developed in partnership with Mayo Clinic
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