| Description |
1 online resource |
| Series |
Handbook of experimental pharmacology ; volume 261 |
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Handbook of experimental pharmacology ; v. 261
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| Contents |
Intro -- Preface -- Contents -- Antiepileptic Drugs in Pediatrics -- 1 Evolution of Anti-seizure Medication Development -- 2 ̀̀First-Generation ́́and ̀̀Second-Generation ́́Antiepileptic Drugs -- 2.1 Phenobarbital -- 2.2 Phenytoin -- 2.3 Ethosuximide -- 2.4 Valproate -- 2.5 Vigabatrin -- 2.6 Zonisamide -- 2.7 Lamotrigine -- 2.8 Felbamate -- 2.9 Topiramate -- 2.10 Oxcarbazepine -- 2.11 Levetiracetam -- 3 Newest-Generation (̀̀Third-Generation)́́ Medications -- 3.1 Pregabalin -- 3.2 Lacosamide -- 3.3 Stiripentol -- 3.4 Rufinamide -- 3.5 Eslicarbazepine Acetate -- 3.6 Perampanel |
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3.7 Brivaracetam -- 3.8 Cannabidiol/Epidiolex -- 3.9 Everolimus -- 4 Drugs in Development -- 4.1 Benzodiazepine Formulations -- 4.2 Bumetanide (NKCC Inhibitor) -- 4.3 Fenfluramine (Amphetamine Derivative) -- 4.4 Ganaxolone (GABAergic) -- 4.5 Huperzine -- 4.6 Ion Channel Modulators -- 4.7 Metabolic Modulators -- References -- Pharmacotherapy of Duchenne Muscular Dystrophy -- 1 Introduction -- 2 Treatment Goals in DMD: Current Landscape -- 3 Treatment Goals in DMD: Pharmacotherapies in the Pipeline -- 4 Treatment Goals in Becker Muscular Dystrophy and Female DMD Carriers -- 5 Looking Forward |
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3.1.1 What Doses and Intervals Are Needed to Restore Normal Enzyme Activity in Relevant Tissues by Intrathecal Injection? -- 3.1.2 Does the Restored Enzyme Activity Reduce Storage Material and Brain Pathology? -- 3.1.3 Do the Drugs Induce Immune Reaction and/or Other Relevant Adverse Reactions? -- 3.1.4 Does Intrathecal ERT Improve the CNS Disease in MPS? -- 3.2 Trojan Horse Approach with Fusion Proteins -- 3.3 Nanotechnology -- 3.4 Gene Therapy -- 3.5 Cell Microencapsulation -- 3.6 Stop Codon Read-Through -- 3.7 Pharmacological Chaperones -- 3.8 GAG-Reducing Small Molecules -- References |
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Current and Emerging Therapies for Mitochondriopathies -- 1 Introduction -- 2 Current Therapies -- 2.1 Improving Electron Transfer Chain (ETC) Function -- 2.2 Energy Buffer -- 2.3 Antioxidants -- 2.4 Restoration of Nitric Oxide Production -- 3 Emerging Therapies -- 3.1 Stimulation of Mitochondrial Biogenesis -- 3.2 Cardiolipin Protection -- 4 Gene Therapy -- References -- GH and IGF-1 Replacement in Children -- 1 Introduction -- 2 Pathophysiology of GHD -- 3 Treatment of GH-Deficient Children with rhGH -- 4 Treatment of Non-GH-Deficient Children with rhGH |
| 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 |
| Bibliography |
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 |
| Subject |
Pediatric pharmacology.
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Children -- Diseases -- Chemotherapy.
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Children.
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Human beings.
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Infants.
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Child
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Humans
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Infant
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children (people by age group)
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Homo sapiens (species)
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infants.
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Paediatric medicine.
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Pharmacology.
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Medical -- Pediatrics.
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Medical -- Pharmacology.
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Children -- Diseases -- Chemotherapy
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Pediatric pharmacology
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| Genre/Form |
Electronic books
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| Form |
Electronic book
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| Author |
Kiess, W. (Wieland)
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Schwab, M. (Matthias)
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Anker, Johannes van den
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| ISBN |
9783030504946 |
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3030504948 |
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