Drug delivery in overcoming the blood-brain barrier: role of nasal mucosal grafting [PDF]
, 2017 The blood–brain barrier (BBB) plays a fundamental role in protecting and maintaining the homeostasis of the brain. For this reason, drug delivery to the brain is much more difficult than that to other compartments of the body. In order to bypass or cross
Carafa, Maria +5 more
core +1 more source
The Impact of Excluding Nonrandomized Studies From Systematic Reviews in Rare Diseases: “The Example of Meta-Analyses Evaluating the Efficacy and Safety of Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis” [PDF]
, 2021 Nonrandomized studies are usually excluded from systematic reviews. This could lead to loss of a considerable amount of information on rare diseases. In this article, we explore the impact of excluding nonrandomized studies on the generalizability of ...
Cortés, Javier +7 more
core +2 more sources
Respiratory and sleep disorders in mucopolysaccharidosis [PDF]
, 2012 MPS encompasses a group of rare lysosomal storage disorders that are associated with the accumulation of glycosaminoglycans (GAG) in organs and tissues.
Ciarán McArdle +9 more
core +1 more source
Enzymatic replacement therapy for lysosomal storage disorders: Drug evaluations review in Spain [PDF]
, 2020 In the European Union companies only need to demonstrate that the risk-benefit balance of the new drug is favourable to obtain the authorization to sell new drugs.
Ascanio, Meritxell, Darbà, Josep
core +1 more source
Active immunotherapy and alternative therapeutic modalities for Alzheimer's disease
Alzheimer's &Dementia: Translational Research &Clinical Interventions, Volume 6, Issue 1, 2020., 2020 Abstract As knowledge of Alzheimer's disease (AD) progression improves, the field has recognized the need to diversify the pipeline, broaden strategies and approaches to therapies, as well as delivery mechanisms. A better understanding of the earliest biological processes of AD/dementia would help inform drug target selection.
Stacie Weninger +12 more
wiley +1 more source
Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance. [PDF]
, 2019 IntroductionMucopolysaccharidosis (MPS) IVA or Morquio A syndrome is an autosomal recessive lysosomal storage disorder (LSD) caused by deficiency of the N-acetylgalactosamine-6-sulfatase (GALNS) enzyme, which impairs lysosomal degradation of keratan ...
Akyol, Mehmet Umut +30 more
core +2 more sources
BioMed Research International, Volume 2020, Issue 1, 2020., 2020 Purpose. Mucopolysaccharidoses (MPS) are group of inherited lysosomal storage diseases caused by mutations of enzymes involved in catalyzing different glycosaminoglycans (GAGs). MPS I and MPS II exhibit both somatic and neurological symptoms with a relatively high disease incidence.
Haiyan Nan +3 more
wiley +1 more source
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy [PDF]
, 2013 For most lysosomal storage diseases (LSDs) affecting the CNS, there is currently no cure. The BBB, which limits the bioavailability of drugs administered systemically, and the short half-life of lysosomal enzymes, hamper the development of effective ...
Albert Ribera +22 more
core +2 more sources
Systematic review of available evidence on 11 high-priced inpatient orphan drugs [PDF]
, 2013 __Background__: Attention for Evidence Based Medicine (EBM) is growing, but evidence for orphan drugs is argued to be limited and inferior. This study systematically reviews the available evidence on clinical effectiveness, costeffectiveness and budget ...
Hakkaart-van Roijen, L. (Leona) +3 more
core +3 more sources
International consensus on (ICON) anaphylaxis [PDF]
, 2014 ICON: Anaphylaxis provides a unique perspective on the principal evidence-based anaphylaxis guidelines developed and published independently from 2010 through 2014 by four allergy/immunology organizations.
Alvarez-Twose +137 more
core +3 more sources