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The Hematology Program is the largest in the country in clinical volumes, with vigorous laboratory and clinical research in sickle cell disease, hemostasis and thrombosis, as well as general hematology, which encompasses red blood cell disorders and rare anemias, thalassemia, platelet disorders, bone marrow failure, immunohematogy, and vascular anomalies.
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Vertex currently operates at the forefront of rare disease scientific innovation and has successfully developed and commercialized multiple breakthrough medicines for Cystic Fibrosis (CF), Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia.
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Extensive comprehension of the healthcare ecosystem including market access landscape and payer reimbursement policies for rare disease products. The Rare Disease Account Specialist is a field-based role responsible for building and managing long-term relationships with physicians and other customers for the accounts in their assigned territory.
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Rare Disease Family Access Manager - Central CA/Inland Empire. Build your own rare disease network/relationships through local advocacy groups, rare disease related events, and attending national conferences.
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Sumitomo Pharma America (SMPA) is focused on delivering therapeutic and scientific breakthroughs in areas of critical patient need spanning psychiatry & neurology, oncology, urology, women's health, rare disease, and cell & gene therapies.
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Additional areas of interest could include computational methods for genome assembly, population genetics, functional genomics, or structural variant interpretation in rare disease. The team member will focus on leading research projects related to analyses of long-read sequencing and structural variation in population studies such as the All of Us Research Program and the genome aggregation database (gnomAD), as well as rare disease programs including the NHGRI Mendelian genomics program (GREGoR) and the NICHD Fetal Genomics Consortium.
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Rare disease or biologics infusion experience. C-Suite roles (CEO, CFO, CBO), VP Finance, VP Clinical Ops, Specialty Pharmacy, Outreach Coordinator, Infusion Site Directors / Enrollers.
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This role will report to the SVP, Head of Therapeutics Development for Moderna and have direct medical responsibility for the clinical development of our Rare Disease Therapies portfolio, including accountable for the overall clinical development plans, innovative clinical studies and protocols, and for ensuring timely execution and medical monitoring all in close collaboration with relevant internal and external pharmaceutical partners, Clinical Research Organizations and regulatory agencies.
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Hires and manages a Business Development team to create and drive business into new and existing accounts within the Cell & Gene Therapy (CGT), Oncology and Rare disease areas. The SVP of Strategic Commercial Development – Cell and Gene Therapy will lead a team of Business Development Representatives and represent commercial leadership for the Cell and Gene Therapy Therapeutic Area as well as provide Business Development oversight for the Neuroscience and Rare Disease groups.
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Areas of focus include therapeutic drugs for autoimmune and rare disease specialty areas like neurology, rheumatology, nephrology, ophthalmology and pulmonology; immunotherapy and neonatal respiratory critical care therapies; and analgesics and central nervous system drugs.
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In this vital role you as Executive Director, Precision Medicine for either Oncology, Cardio-metabolic or Inflammation/rare disease, you will partner with specific Therapeutic Area leads across Research & Development, guiding the development of translational, reverse translational, and precision medicine strategies.
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Team Contribution – Lead Pfizer's Rare Disease portfolio in the Sickle Cell Centers and Clinics within the territory. Effectively utilize marketing materials, programs, and other company offerings and resources to educate customers, build meaningful relationships, and drive performance within the Rare Disease Sickle Cell portfolio.
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Prior experience in data andanalyticsfor specialty medicine/biologics/rare disease and Oncology will be a significant asset. At least 10 years of extensive experience and deep understanding of different types of syndicatedcommercialpharmadatasets across Claims, Retail Sales, Non-retail Sales, Trade data, Specialty Pharmacy (SP/SD), Hub, Formulary, Co-pay, Prescriber-Plan data, EMR/EHR, Diagnostic data, Shipment, Inventory, Non-personal Promotion data, Speaker Programs, Email Campaigns, Digital Data (HCP Portal, Patient Portal, Brand websites, Third-party website), Conferences etc.
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Vertex currently operates at the forefront of rare disease scientific innovation and has successfully developed and commercialized multiple breakthrough medicines for Cystic Fibrosis (CF) and Sickle Cell Disease.
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The MSL position is to provide educational, scientific, and research support for the SOBI rare disease portfolio of products in the hematology franchise. Disease state knowledge, hematology, and rare disease preferred.
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