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Availability, Accessibility and Delivery to Patients of the 28 Orphan Medicines Approved by the European Medicine Agency for Hereditary Metabolic Diseases in the MetabERN Network
Abstract: Background The European Medicine Agency granted marketing approval to 164 orphan medicinal products for rare diseases, among which 28 products intended for the treatment of hereditary metabolic diseases. Taking advantage of its privileged connection with 69 healthcare centres of excellence in this field, MetabERN, the European Reference Network for hereditary metabolic diseases, performed a survey asking health care providers from 18 European countries whether these products are available on the market, reimbursed and therefore accessible for prescription, and actually delivered in their centre. Results Responses received from 52 centres (75%) concerned the design of treatment plan...
Nanoarchitectonics for Brain Drug Delivery
This book discusses basics of brain diseases and the role of nanobiotechnology in existing treatment options for neurodegenerative disorders. It begins with an overview of brain diseases and the need for novel drug-delivery approaches. It highlights the current route for the intranasal advanced drug-delivery systems for brain diseases. It also discusses innovative categories of drug-delivery systems, including mesoporous silica nanoparticles, polymeric nanocarriers, and lipid-based nanocarriers through multi-responsive DDSs and their implications in brain disorders. Features: Includes an overview of brain diseases and highlights the need for novel drug-delivery approaches Focuses on theoreti...
Lysosomal Storage Disorders
Lysosomal Storage Disorders Enables readers to gain both holistic and specific knowledge on the topic of Lysosomal Storage Disorders In the past few decades, we have witnessed a ‘golden age’ of Lysosomal Storage Disorders (LSDs) clinical care, diagnosis, and research and this book is a well-timed review of the rapidly developing subject of LSDs. Contributions from many leading scientists and clinicians in the field provide the reader with a completely comprehensive overview of the subject. Topics covered in the book include: The general aspects of LSDs, with special attention paid to physiology and pathology Clinical and laboratory diagnosis, including newborn screening and the genetics ...
Nanomedicine and Neurosciences: Advantages, Limitations and Safety Aspects
Frontiers in Nanomedicine offers an up-to-date understanding of nanomaterials to readers having clinical or biomolecular research interests. Scientists, both aspiring and experienced, will find, in each volume, a comprehensive overview of current molecular strategies for using nanoscale materials in medicine. Nanomedicine and Neurosciences: Advantages, Limitations and Safety Aspects presents different aspects of nanomedicine applied to neuroscience for the diagnosis of disease and the role of nanoparticles in targeted drug delivery systems for neurodegenerative disorders. Topics covered in this volume cover the physiology of neurodegeneration, targeted therapies for Alzheimer’s disease and Parkinson’s disease, blood brain barrier drug delivery systems, in vivo studies of drug nanoconjugates, nanomedicine safety, and more.
Respiratory Infections in Allergy and Asthma
This reference presents investigations into the role of respiratory infections in protecting against allergy and asthma development, the activation of airway inflammation, and exacerbations of asthma--exploring cellular and molecular mechanisms, as well as factors including genetics, the environment, and allergen exposure.
Lysosomal Storage Diseases
The last fifteen years have witnessed the extraordinary evolution of basic and clinical research in the field of lysoso-mal storage diseases (LSDs), transforming many of them from dire, untreatable progressive diseases to conditions that allow for possible cure or mitigation. In addition to the presently employed techniques of haematopoietic stem cell transplantation and enzyme replacement for a number of lysoso-mal storage diseases, other therapeutic approaches are being developed that are based on different principles. The awareness that the efficacy of treatment is greater if adminis-tered at the first signs of disease or, even better, during the pre-symptomatic phase underscores the urge...
Neurodegenerative Diseases: Integrative PPPM Approach as the Medicine of the Future
This book will compile a collection of chapters dedicated to varied aspects of PPPM in neuropsychiatric and neurodegenerative diseases. Among the topics to be covered are: Recent advances in ALS research News about Clinical aspects and advanced therapy approaches in personalized treatment of ALS Schizophrenia: New treatments and clinical aspects Predictive, Preventive and Personalised Medicine in aging macular degeneration Advances in Multiple Sclerosis Pharmacogenetics, Tailoring Treatment Efficacy, Safety and Regimen Selection Multiple sclerosis related biomarkers: perspectives for clinical application Preventive clinical trials in brain aging: new trends & the need of guidelines MCI_ clinical guidelines in early diagnosis of dementia Alzheimer's disease: diagnostics, prognostics and the road to prevention Biomarkers for early diagnosis of Parkinson's and Alzheimer's diseases Synucleinopathies, tauopathies, TDP-43 proteinopathies and amyloidosis PSP, MSA and other parkinsonisms
