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The development of paediatric medicines can be challenging since this is a different patient population with specific needs. A medicine designed for use in paediatric patients must consider the following aspects: patient population variability; the need for dose flexibility; route of administration; patient compliance; excipient tolerability. For example, the toxicity of excipients may differ in children compared to adults and children have different taste preferences. Globally, about 75% of drugs do not carry regulatory approval for use in children; worldwide, many medications prescribed for the treatment of paediatric diseases are used off-label, and less than 20% of package inserts have sufficient information for treating children. This book provides an update on both state-of-the-art methodology and operational challenges in paediatric formulation design and development. It aims at re-evaluating what is needed for more progress in the design and development of age-appropriate treatments for paediatric diseases, focusing on: formulation development; drug delivery design; efficacy, safety, and tolerability of drugs and excipients.
Medicine --- dasatinib --- Duchenne muscular dystrophy --- cyclodextrin inclusion complex --- phase solubility studies --- paediatric age --- liquid formulation --- tumorspheres --- retinoblastoma --- topotecan --- penetration --- confocal microscopy --- poorly water soluble drug --- solubility enhancement --- grinding --- spray congealing --- neglected tropical diseases --- polymorph --- Norvir® --- ritonavir --- poorly soluble compound --- pediatric --- palatability assessment --- bioavailability --- flavor profile --- Interleukin-1 --- anakinra --- canakinumab --- innovative biotechnologies --- autoinflammatory disease --- Kawasaki disease --- systemic juvenile idiopathic arthritis --- personalized medicine --- child --- pediatrics --- neonates --- formulation --- product development --- formulation development --- oral --- parenteral --- topical --- inhaled --- intra nasal --- biopharmaceutics --- administration --- excipient --- NICU --- device --- medication error --- dosage form --- modified release --- drug delivery --- paediatric formulation development --- paediatric dosage forms --- chronic myeloid leukemia --- tyrosine kinase inhibitors --- pediatric age --- imatinib --- nilotinb --- ponatinib --- Orodispersible formulation --- pyrazinamide --- pediatric drug delivery --- tuberculosis --- design of experiments --- children --- edible films --- development --- design --- paediatric --- age-related --- palatable --- taste-masking --- acceptable --- dasatinib --- Duchenne muscular dystrophy --- cyclodextrin inclusion complex --- phase solubility studies --- paediatric age --- liquid formulation --- tumorspheres --- retinoblastoma --- topotecan --- penetration --- confocal microscopy --- poorly water soluble drug --- solubility enhancement --- grinding --- spray congealing --- neglected tropical diseases --- polymorph --- Norvir® --- ritonavir --- poorly soluble compound --- pediatric --- palatability assessment --- bioavailability --- flavor profile --- Interleukin-1 --- anakinra --- canakinumab --- innovative biotechnologies --- autoinflammatory disease --- Kawasaki disease --- systemic juvenile idiopathic arthritis --- personalized medicine --- child --- pediatrics --- neonates --- formulation --- product development --- formulation development --- oral --- parenteral --- topical --- inhaled --- intra nasal --- biopharmaceutics --- administration --- excipient --- NICU --- device --- medication error --- dosage form --- modified release --- drug delivery --- paediatric formulation development --- paediatric dosage forms --- chronic myeloid leukemia --- tyrosine kinase inhibitors --- pediatric age --- imatinib --- nilotinb --- ponatinib --- Orodispersible formulation --- pyrazinamide --- pediatric drug delivery --- tuberculosis --- design of experiments --- children --- edible films --- development --- design --- paediatric --- age-related --- palatable --- taste-masking --- acceptable
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Autoinflammation, as a relatively new field in clinical rheumatology, has gained an increasing importance in recent years. The number of identified entities and affected patients has gradually increased, and some of the involved pathways have already been identified. This progress allows a deeper understanding of closely linked diseases, namely, inflammasomopathies, interferonopathies, Relo-pathies, and proteasome associated syndromes. These insights have not only improved their classification but also helped to identify new treatment targets of pro-inflammatory cytokines, including IL-1ß, IL-6, interferon-, and TNF-alpha. Nevertheless, there is still a high medical need, especially in reliable outcome measures, for confirmation of data from controlled clinical trials and, finally, also for long-term experience from registers. This issue welcomes all types of papers on the broad spectrum of clinical characteristics, prognosis, pathophysiology, and treatment of autoinflammatory diseases. The goal of this Special Issue is to further raise awareness of autoinflammatory processes and to better separate them from well-established autoimmune diseases. It is clear that we have entered a new age in this complex field, linking rheumatology even closer to immunology.
gout --- febuxostat --- colchicine --- hepatotoxicity --- prophylaxis --- myositis --- inflammatory idiopathic myopathy --- dysphagia --- aspiration --- pneumonia --- immunoglobulin G4-related orbital disease (IgG4-ROD) --- orbital lymphoma (OL) --- computed tomography (CT) --- Hounsfield unit --- imaging --- autoinflammation --- arthritis --- CAPS --- FCAS --- MWS --- CINCA --- NOMID --- hearing loss --- urticarial-like rash --- autoinflammatory disease --- anti-IL-1 treatment --- rheumatoid arthritis --- synovitis --- neoplasms --- edema --- inflammation --- new genetic variant --- monogenic autoinflammatory syndrome --- diagnostic delay --- anakinra --- damage index --- genetic inheritance --- personalized therapy --- Interleukin-1 --- autoinflammatory diseases --- FMF --- coronavirus --- SARS-CoV-2 antibody response --- adult-onset Still’s disease --- autoinflammatory disorder --- systemic-onset juvenile idiopathic arthritis --- haemophagocytic lymphohistiocytosis --- macrophage activation syndrome --- IFN-γ --- JAK inhibitor --- proliferation --- DNA damage repair --- γH2AX --- PBMCs --- T lymphocytes --- proteasome --- autoimmune --- proteasome-associated autoinflammatory syndrome --- therapy --- IL-1 inhibitors --- NGS --- SURF --- spondyloarthritis --- human leukocyte antigen --- undifferentiated enthesitis and/or arthritis --- ASAS classification criteria --- clinical management --- canakinumab --- cytokines --- n/a --- adult-onset Still's disease
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Skin inflammation is associated with a wide range of conditions which represent major health issues worldwide. Skin and mucosal surfaces represent the primary interface between the human body and the environment, susceptible to numerous factors whose action results in diseases produced by chemical substances, mechanical trauma, microbial agents, radiation, etc. Inflammation, a complex network of interactions between soluble molecules and cells, represents the main modality of the skin’s response to injuries. Numerous studies have revealed close links between chronic inflammation, oxidative stress, and carcinogenesis. Chronic inflammation induces the activation of various cell types and an increase in the production of reactive oxygen species, promoting the initiation of a malignant process. Identifying specific biomarkers is essential for understanding molecular mechanisms and developing therapies appropriate to the patient’s characteristics.Personalized medicine is an emerging field of medicine that has the potential to predict which therapy will be safe and efficacious for specific patients using an individual’s genetic profile to guide decisions regarding the diagnosis, treatment, as well as prevention of disease. This book gathers articles that present recent advancements in research involving the mechanisms that underlie the development of inflammatory skin disorders, skin and mucosal inflammation in general.
sea-buckthorn seed oil --- long-chain fatty acids --- skin dysplastic keratinocytes --- UVA --- CD36 --- SR-B2 --- atopic dermatitis --- eczema --- JAK inhibitors --- systematic review --- meta-analysis --- evidence-based medicine --- immune-mediated skin diseases --- target therapy --- skin conditions and systemic inflammatory diseases --- systemic sclerosis --- biomarker --- calumenin --- S100A6 --- cytohesin 2 --- cannabinoids --- inflammation --- gut-lung-skin barrier --- signaling pathways --- inflammatory biomarkers --- psoriasis vulgaris --- prevalence --- comorbidities --- risk factors --- systemic lupus erythematous --- lupus nephritis --- lipid peroxidation --- DNA oxidation --- oxidized proteins --- carbohydrate oxidation --- antioxidative stress strategies --- biomarkers --- IgY --- psoriatic dermatitis --- imiquimod --- C57 BL/6 mice --- alginate --- biomaterial --- dressing --- fibers --- hydrogel --- nanofibers --- commercially available --- wound care --- wound healing --- cutaneous melanoma --- epigenetic regulation --- drug resistance --- therapeutic targets --- epigenetic therapy --- immune response --- Sutton nevi --- halo nevi --- skin tumor --- psoriasis --- proteome analysis --- estrogen --- menopause --- cSCC --- AK --- sialylation --- sialyltransferase --- sialidase --- COVID-19 --- pyoderma gangrenosum --- immunosuppression --- cyclosporine --- corticosteroids --- autoinflammatory disease --- androgenic alopecia --- clinical trial --- hair density --- platelet-rich plasma --- n/a
Choose an application
The development of paediatric medicines can be challenging since this is a different patient population with specific needs. A medicine designed for use in paediatric patients must consider the following aspects: patient population variability; the need for dose flexibility; route of administration; patient compliance; excipient tolerability. For example, the toxicity of excipients may differ in children compared to adults and children have different taste preferences. Globally, about 75% of drugs do not carry regulatory approval for use in children; worldwide, many medications prescribed for the treatment of paediatric diseases are used off-label, and less than 20% of package inserts have sufficient information for treating children. This book provides an update on both state-of-the-art methodology and operational challenges in paediatric formulation design and development. It aims at re-evaluating what is needed for more progress in the design and development of age-appropriate treatments for paediatric diseases, focusing on: formulation development; drug delivery design; efficacy, safety, and tolerability of drugs and excipients.
dasatinib --- Duchenne muscular dystrophy --- cyclodextrin inclusion complex --- phase solubility studies --- paediatric age --- liquid formulation --- tumorspheres --- retinoblastoma --- topotecan --- penetration --- confocal microscopy --- poorly water soluble drug --- solubility enhancement --- grinding --- spray congealing --- neglected tropical diseases --- polymorph --- Norvir® --- ritonavir --- poorly soluble compound --- pediatric --- palatability assessment --- bioavailability --- flavor profile --- Interleukin-1 --- anakinra --- canakinumab --- innovative biotechnologies --- autoinflammatory disease --- Kawasaki disease --- systemic juvenile idiopathic arthritis --- personalized medicine --- child --- pediatrics --- neonates --- formulation --- product development --- formulation development --- oral --- parenteral --- topical --- inhaled --- intra nasal --- biopharmaceutics --- administration --- excipient --- NICU --- device --- medication error --- dosage form --- modified release --- drug delivery --- paediatric formulation development --- paediatric dosage forms --- chronic myeloid leukemia --- tyrosine kinase inhibitors --- pediatric age --- imatinib --- nilotinb --- ponatinib --- Orodispersible formulation --- pyrazinamide --- pediatric drug delivery --- tuberculosis --- design of experiments --- children --- edible films --- development --- design --- paediatric --- age-related --- palatable --- taste-masking --- acceptable
Choose an application
Skin inflammation is associated with a wide range of conditions which represent major health issues worldwide. Skin and mucosal surfaces represent the primary interface between the human body and the environment, susceptible to numerous factors whose action results in diseases produced by chemical substances, mechanical trauma, microbial agents, radiation, etc. Inflammation, a complex network of interactions between soluble molecules and cells, represents the main modality of the skin’s response to injuries. Numerous studies have revealed close links between chronic inflammation, oxidative stress, and carcinogenesis. Chronic inflammation induces the activation of various cell types and an increase in the production of reactive oxygen species, promoting the initiation of a malignant process. Identifying specific biomarkers is essential for understanding molecular mechanisms and developing therapies appropriate to the patient’s characteristics.Personalized medicine is an emerging field of medicine that has the potential to predict which therapy will be safe and efficacious for specific patients using an individual’s genetic profile to guide decisions regarding the diagnosis, treatment, as well as prevention of disease. This book gathers articles that present recent advancements in research involving the mechanisms that underlie the development of inflammatory skin disorders, skin and mucosal inflammation in general.
Medicine --- sea-buckthorn seed oil --- long-chain fatty acids --- skin dysplastic keratinocytes --- UVA --- CD36 --- SR-B2 --- atopic dermatitis --- eczema --- JAK inhibitors --- systematic review --- meta-analysis --- evidence-based medicine --- immune-mediated skin diseases --- target therapy --- skin conditions and systemic inflammatory diseases --- systemic sclerosis --- biomarker --- calumenin --- S100A6 --- cytohesin 2 --- cannabinoids --- inflammation --- gut-lung-skin barrier --- signaling pathways --- inflammatory biomarkers --- psoriasis vulgaris --- prevalence --- comorbidities --- risk factors --- systemic lupus erythematous --- lupus nephritis --- lipid peroxidation --- DNA oxidation --- oxidized proteins --- carbohydrate oxidation --- antioxidative stress strategies --- biomarkers --- IgY --- psoriatic dermatitis --- imiquimod --- C57 BL/6 mice --- alginate --- biomaterial --- dressing --- fibers --- hydrogel --- nanofibers --- commercially available --- wound care --- wound healing --- cutaneous melanoma --- epigenetic regulation --- drug resistance --- therapeutic targets --- epigenetic therapy --- immune response --- Sutton nevi --- halo nevi --- skin tumor --- psoriasis --- proteome analysis --- estrogen --- menopause --- cSCC --- AK --- sialylation --- sialyltransferase --- sialidase --- COVID-19 --- pyoderma gangrenosum --- immunosuppression --- cyclosporine --- corticosteroids --- autoinflammatory disease --- androgenic alopecia --- clinical trial --- hair density --- platelet-rich plasma --- sea-buckthorn seed oil --- long-chain fatty acids --- skin dysplastic keratinocytes --- UVA --- CD36 --- SR-B2 --- atopic dermatitis --- eczema --- JAK inhibitors --- systematic review --- meta-analysis --- evidence-based medicine --- immune-mediated skin diseases --- target therapy --- skin conditions and systemic inflammatory diseases --- systemic sclerosis --- biomarker --- calumenin --- S100A6 --- cytohesin 2 --- cannabinoids --- inflammation --- gut-lung-skin barrier --- signaling pathways --- inflammatory biomarkers --- psoriasis vulgaris --- prevalence --- comorbidities --- risk factors --- systemic lupus erythematous --- lupus nephritis --- lipid peroxidation --- DNA oxidation --- oxidized proteins --- carbohydrate oxidation --- antioxidative stress strategies --- biomarkers --- IgY --- psoriatic dermatitis --- imiquimod --- C57 BL/6 mice --- alginate --- biomaterial --- dressing --- fibers --- hydrogel --- nanofibers --- commercially available --- wound care --- wound healing --- cutaneous melanoma --- epigenetic regulation --- drug resistance --- therapeutic targets --- epigenetic therapy --- immune response --- Sutton nevi --- halo nevi --- skin tumor --- psoriasis --- proteome analysis --- estrogen --- menopause --- cSCC --- AK --- sialylation --- sialyltransferase --- sialidase --- COVID-19 --- pyoderma gangrenosum --- immunosuppression --- cyclosporine --- corticosteroids --- autoinflammatory disease --- androgenic alopecia --- clinical trial --- hair density --- platelet-rich plasma
Choose an application
Autoinflammation, as a relatively new field in clinical rheumatology, has gained an increasing importance in recent years. The number of identified entities and affected patients has gradually increased, and some of the involved pathways have already been identified. This progress allows a deeper understanding of closely linked diseases, namely, inflammasomopathies, interferonopathies, Relo-pathies, and proteasome associated syndromes. These insights have not only improved their classification but also helped to identify new treatment targets of pro-inflammatory cytokines, including IL-1ß, IL-6, interferon-, and TNF-alpha. Nevertheless, there is still a high medical need, especially in reliable outcome measures, for confirmation of data from controlled clinical trials and, finally, also for long-term experience from registers. This issue welcomes all types of papers on the broad spectrum of clinical characteristics, prognosis, pathophysiology, and treatment of autoinflammatory diseases. The goal of this Special Issue is to further raise awareness of autoinflammatory processes and to better separate them from well-established autoimmune diseases. It is clear that we have entered a new age in this complex field, linking rheumatology even closer to immunology.
Medicine --- gout --- febuxostat --- colchicine --- hepatotoxicity --- prophylaxis --- myositis --- inflammatory idiopathic myopathy --- dysphagia --- aspiration --- pneumonia --- immunoglobulin G4-related orbital disease (IgG4-ROD) --- orbital lymphoma (OL) --- computed tomography (CT) --- Hounsfield unit --- imaging --- autoinflammation --- arthritis --- CAPS --- FCAS --- MWS --- CINCA --- NOMID --- hearing loss --- urticarial-like rash --- autoinflammatory disease --- anti-IL-1 treatment --- rheumatoid arthritis --- synovitis --- neoplasms --- edema --- inflammation --- new genetic variant --- monogenic autoinflammatory syndrome --- diagnostic delay --- anakinra --- damage index --- genetic inheritance --- personalized therapy --- Interleukin-1 --- autoinflammatory diseases --- FMF --- coronavirus --- SARS-CoV-2 antibody response --- adult-onset Still's disease --- autoinflammatory disorder --- systemic-onset juvenile idiopathic arthritis --- haemophagocytic lymphohistiocytosis --- macrophage activation syndrome --- IFN-γ --- JAK inhibitor --- proliferation --- DNA damage repair --- γH2AX --- PBMCs --- T lymphocytes --- proteasome --- autoimmune --- proteasome-associated autoinflammatory syndrome --- therapy --- IL-1 inhibitors --- NGS --- SURF --- spondyloarthritis --- human leukocyte antigen --- undifferentiated enthesitis and/or arthritis --- ASAS classification criteria --- clinical management --- canakinumab --- cytokines --- gout --- febuxostat --- colchicine --- hepatotoxicity --- prophylaxis --- myositis --- inflammatory idiopathic myopathy --- dysphagia --- aspiration --- pneumonia --- immunoglobulin G4-related orbital disease (IgG4-ROD) --- orbital lymphoma (OL) --- computed tomography (CT) --- Hounsfield unit --- imaging --- autoinflammation --- arthritis --- CAPS --- FCAS --- MWS --- CINCA --- NOMID --- hearing loss --- urticarial-like rash --- autoinflammatory disease --- anti-IL-1 treatment --- rheumatoid arthritis --- synovitis --- neoplasms --- edema --- inflammation --- new genetic variant --- monogenic autoinflammatory syndrome --- diagnostic delay --- anakinra --- damage index --- genetic inheritance --- personalized therapy --- Interleukin-1 --- autoinflammatory diseases --- FMF --- coronavirus --- SARS-CoV-2 antibody response --- adult-onset Still's disease --- autoinflammatory disorder --- systemic-onset juvenile idiopathic arthritis --- haemophagocytic lymphohistiocytosis --- macrophage activation syndrome --- IFN-γ --- JAK inhibitor --- proliferation --- DNA damage repair --- γH2AX --- PBMCs --- T lymphocytes --- proteasome --- autoimmune --- proteasome-associated autoinflammatory syndrome --- therapy --- IL-1 inhibitors --- NGS --- SURF --- spondyloarthritis --- human leukocyte antigen --- undifferentiated enthesitis and/or arthritis --- ASAS classification criteria --- clinical management --- canakinumab --- cytokines
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