Hidden Bibliographic Details
| Other authors / contributors: | Kiess, W. (Wieland)
Schwab, M. (Matthias)
Anker, Johannes van den.
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| ISBN: | 9783030504946
3030504948
303050493X
9783030504939
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| Notes: | References -- Current and Emerging Therapies for Mucopolysaccharidoses -- 1 Introduction -- 2 Current Therapies -- 2.1 Pharmaceutical Characteristics of ERT Medicines -- 2.2 Pharmacology of ERT Medicines -- 2.3 Effects and Limitations of ERT and BMT/HSCT -- 2.3.1 Immunoreactivity -- 2.3.2 Low Bioavailability in Certain Tissues -- 2.3.3 Short-Term Exposure -- 2.3.4 Late Initiation of Treatment -- 3 Emerging Therapies -- 3.1 Intrathecal Enzyme Replacement in MPS I, MPS II, MPS IIIA, and MPS IIIB
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| Summary: | This volume provides readers with the most updated scientific information on the efficacy and safety of medicines for children and adolescents. The book enriches the understanding of pediatric pharmacotherapy for health professionals, regulatory agencies, pharmaceutical companies and learned societies. It contains important information on the pharmacodynamics and pharmacokinetics of drugs. It summarizes the latest investigations on the effects of pharmacological treatments in relation to and dependent on age, gender, fat mass and disease status. Therefore and importantly, this volume reviews the latest data on how pharmacotherapy has to be adjusted and personalized in regards to stages of development and during the pediatric lifespan from neonate through adolescence. In addition, the topic of rare diseases and special challenges for pharmacotherapy will be included and will provide readers with the necessary knowledge to handle complex diseases and treatment strategies especially in relation to pharmacotherapy of rare and orphan diseases.
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| Other form: | Print version: 303050493X 9783030504939
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| Standard no.: | 10.1007/978-3-030-50
10.1007/978-3-030-50494-6
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