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Canadian National Pancreas Conference 2023: A Review of Multidisciplinary Engagement in Pancreatic Cancer Care.
- Published in:
- Current Oncology, 2024, v. 31, n. 10, p. 6191, doi. 10.3390/curroncol31100461
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- Publication type:
- Article
Dirichlet latent modelling enables effective learning and sampling of the functional protein design space.
- Published in:
- Nature Communications, 2024, v. 15, n. 1, p. 1, doi. 10.1038/s41467-024-53622-6
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- Publication type:
- Article
Overall assessment of patients with type 1 Gaucher disease: a single-centre's experience.
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- Journal of Rare Diseases, 2023, v. 2, n. 1, p. 1, doi. 10.1007/s44162-023-00019-6
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- Publication type:
- Article
Cervical spondylolisthesis in mucopolysaccharidosis type II.
- Published in:
- Neurological Sciences, 2023, v. 44, n. 1, p. 409, doi. 10.1007/s10072-022-06357-5
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- Publication type:
- Article
Nanoformulation of Therapeutic Enzymes: A Short Review.
- Published in:
- Periodica Polytechnica: Chemical Engineering, 2023, v. 67, n. 4, p. 624, doi. 10.3311/PPch.22826
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- Publication type:
- Article
P6‐28: Clinical case of using intravenous forms of anti‐tuberculosis drugs to improve the treatment efficiency of tuberculosis in patients with malabsorption syndrome (MS).
- Published in:
- Respirology, 2021, v. 26, p. 237, doi. 10.1111/resp.14150_394
- Publication type:
- Article
P6‐28: Clinical case of using intravenous forms of anti‐tuberculosis drugs to improve the treatment efficiency of tuberculosis in patients with malabsorption syndrome (MS).
- Published in:
- Respirology, 2021, v. 26, p. 237, doi. 10.1111/resp.14150_394
- Publication type:
- Article
Upregulation of Cytotoxic T-cells in pediatric patients with Gaucher disease.
- Published in:
- Scientific Reports, 2022, v. 12, n. 1, p. 1, doi. 10.1038/s41598-022-08843-4
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- Publication type:
- Article
Evaluation of alveolar bone hypomineralization in pediatric hypophosphatasia using orthopantomography.
- Published in:
- Scientific Reports, 2022, v. 12, n. 1, p. 1, doi. 10.1038/s41598-022-05171-5
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- Publication type:
- Article
Author Correction: Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy.
- Published in:
- 2021
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- Publication type:
- Correction Notice
Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy.
- Published in:
- Scientific Reports, 2021, v. 11, n. 1, p. 1, doi. 10.1038/s41598-021-02572-w
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- Publication type:
- Article
Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy.
- Published in:
- Scientific Reports, 2021, v. 11, n. 1, p. 1, doi. 10.1038/s41598-021-02572-w
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- Publication type:
- Article
Early Symptoms and Treatment Outcomes in Neuronal Ceroid Lipofuscinosis Type 2: Croatian Experience.
- Published in:
- Journal of Personalized Medicine, 2024, v. 14, n. 8, p. 783, doi. 10.3390/jpm14080783
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- Publication type:
- Article
Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis.
- Published in:
- Journal of Personalized Medicine, 2022, v. 12, n. 8, p. 1338, doi. 10.3390/jpm12081338
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- Publication type:
- Article
Newborn Screening Program for Mucopolysaccharidosis Type II and Long-Term Follow-Up of the Screen-Positive Subjects in Taiwan.
- Published in:
- Journal of Personalized Medicine, 2022, v. 12, n. 7, p. 1023, doi. 10.3390/jpm12071023
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- Publication type:
- Article
Individualized Assessment of Exercise Capacity in Response to Acute and Long-Term Enzyme Replacement Therapy in Pediatric Pompe Disease.
- Published in:
- Journal of Personalized Medicine, 2021, v. 11, n. 11, p. 1105, doi. 10.3390/jpm11111105
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- Publication type:
- Article
Migalastat Tissue Distribution: Extrapolation From Mice to Humans Using Pharmacokinetic Modeling and Comparison With Agalsidase Beta Tissue Distribution in Mice.
- Published in:
- Clinical Pharmacology in Drug Development, 2021, v. 10, n. 9, p. 1075, doi. 10.1002/cpdd.941
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- Publication type:
- Article
Therapeutic Strategies For Tay-Sachs Disease.
- Published in:
- Frontiers in Pharmacology, 2022, v. 13, p. 1, doi. 10.3389/fphar.2022.906647
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- Publication type:
- Article
A Multi-Centre Prospective Study of the Efficacy and Safety of Alglucosidase Alfa in Chinese Patients With Infantile-Onset Pompe Disease.
- Published in:
- Frontiers in Pharmacology, 2022, v. 13, p. 1, doi. 10.3389/fphar.2022.903488
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- Publication type:
- Article
The Inflammation in the Cytopathology of Patients With Mucopolysaccharidoses- Immunomodulatory Drugs as an Approach to Therapy.
- Published in:
- Frontiers in Pharmacology, 2022, v. 13, p. 1, doi. 10.3389/fphar.2022.863667
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- Publication type:
- Article
Evidence of Lysosomal β-Hexosaminidase Enzymatic Activity Associated with Extracellular Vesicles: Potential Applications for the Correction of Sandhoff Disease.
- Published in:
- Journal of Functional Biomaterials, 2024, v. 15, n. 6, p. 153, doi. 10.3390/jfb15060153
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- Publication type:
- Article
Role of the Lactide:Glycolide Ratio in PLGA Nanoparticle Stability and Release under Lysosomal Conditions for Enzyme Replacement Therapy of Lysosomal Storage Disorders.
- Published in:
- Journal of Functional Biomaterials, 2023, v. 14, n. 9, p. 440, doi. 10.3390/jfb14090440
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- Publication type:
- Article
HexA-Enzyme Coated Polymer Nanoparticles for the Development of a Drug-Delivery System in the Treatment of Sandhoff Lysosomal Storage Disease.
- Published in:
- Journal of Functional Biomaterials, 2022, v. 13, n. 2, p. 37, doi. 10.3390/jfb13020037
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- Publication type:
- Article
The Safety of Agalsidase Alfa Enzyme Replacement Therapy in Canadian Patients with Fabry Disease Following Implementation of a Bioreactor Process.
- Published in:
- Drugs in R&D, 2021, v. 21, n. 4, p. 385, doi. 10.1007/s40268-021-00361-4
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- Publication type:
- Article
Decreased Levels of Chaperones in Mucopolysaccharidoses and Their Elevation as a Putative Auxiliary Therapeutic Approach.
- Published in:
- Pharmaceutics, 2023, v. 15, n. 2, p. 704, doi. 10.3390/pharmaceutics15020704
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- Publication type:
- Article
Current and Future Therapeutic Approaches of Exocrine Pancreatic Insufficiency in Children with Cystic Fibrosis in the Era of Personalized Medicine.
- Published in:
- Pharmaceutics, 2023, v. 15, n. 1, p. 162, doi. 10.3390/pharmaceutics15010162
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- Publication type:
- Article
Treatment of Neuronopathic Mucopolysaccharidoses with Blood–Brain Barrier-Crossing Enzymes: Clinical Application of Receptor-Mediated Transcytosis.
- Published in:
- Pharmaceutics, 2022, v. 14, n. 6, p. 1240, doi. 10.3390/pharmaceutics14061240
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- Publication type:
- Article
Comparative Investigation of pH–Dependent Availability of Pancreatic Enzyme Preparations In Vitro.
- Published in:
- Pharmaceuticals (14248247), 2024, v. 17, n. 5, p. 552, doi. 10.3390/ph17050552
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- Publication type:
- Article
Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease.
- Published in:
- 2023
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- Publication type:
- Case Study
Hydrogel Delivery Device for the In Vitro and In Vivo Sustained Release of Active rhGALNS Enzyme.
- Published in:
- Pharmaceuticals (14248247), 2023, v. 16, n. 7, p. 931, doi. 10.3390/ph16070931
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- Publication type:
- Article
Treatment of Fabry Disease: Established and Emerging Therapies.
- Published in:
- Pharmaceuticals (14248247), 2023, v. 16, n. 2, p. 320, doi. 10.3390/ph16020320
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- Publication type:
- Article
Effect of Asfotase Alfa in the Treatment of Hypophosphatasia- A Systematic Review.
- Published in:
- Journal of Pharmacy & Bioallied Sciences, 2023, v. 15, p. S101, doi. 10.4103/jpbs.jpbs_662_22
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- Publication type:
- Article
Enzyme Replacement Therapy for Succinic Semialdehyde Dehydrogenase Deficiency: Relevance in γ-Aminobutyric Acid Plasticity.
- Published in:
- Journal of Child Neurology, 2021, v. 36, n. 13/14, p. 1200, doi. 10.1177/0883073821993000
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- Publication type:
- Article
Proceedings of the International SSADH Deficiency 2020 Conference.
- Published in:
- 2021
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- Publication type:
- Editorial
Development of the "Hamburg Best Practice Guidelines for ICV−Enzyme Replacement therapy (ERT) in CLN2 Disease" Based on 6 Years Treatment Experience in 48 Patients.
- Published in:
- Journal of Child Neurology, 2021, v. 36, n. 8, p. 635, doi. 10.1177/0883073821989154
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- Publication type:
- Article
Treatment with Polyethylene Glycol–Conjugated Fungal d-Amino Acid Oxidase Reduces Lung Inflammation in a Mouse Model of Chronic Granulomatous Disease.
- Published in:
- Inflammation, 2022, v. 45, n. 4, p. 1668, doi. 10.1007/s10753-022-01650-z
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- Publication type:
- Article
Assessment of the function and morphology of the thyroid gland in paediatric patients treated with enzyme replacement therapy due to selected storage diseases - preliminary results of our own research and a review of the literature.
- Published in:
- Pediatric Endocrinology, Diabetes & Metabolism, 2022, v. 28, n. 2, p. 114, doi. 10.5114/pedm.2022.112860
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- Publication type:
- Article
Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial.
- Published in:
- Brain: A Journal of Neurology, 2023, v. 146, n. 2, p. 461, doi. 10.1093/brain/awac379
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- Publication type:
- Article
High-Dose Ambroxol Therapy in Type 1 Gaucher Disease Focusing on Patients with Poor Response to Enzyme Replacement Therapy or Substrate Reduction Therapy.
- Published in:
- International Journal of Molecular Sciences, 2023, v. 24, n. 7, p. 6732, doi. 10.3390/ijms24076732
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- Publication type:
- Article
Enzyme Replacement Therapy for FABRY Disease: Possible Strategies to Improve Its Efficacy.
- Published in:
- International Journal of Molecular Sciences, 2023, v. 24, n. 5, p. 4548, doi. 10.3390/ijms24054548
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- Publication type:
- Article
Contribution of Glucosylsphingosine (Lyso-Gb1) to Treatment Decisions in Patients with Gaucher Disease.
- Published in:
- International Journal of Molecular Sciences, 2023, v. 24, n. 4, p. 3945, doi. 10.3390/ijms24043945
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- Publication type:
- Article
Mucopolysaccharidoses: Cellular Consequences of Glycosaminoglycans Accumulation and Potential Targets.
- Published in:
- International Journal of Molecular Sciences, 2023, v. 24, n. 1, p. 477, doi. 10.3390/ijms24010477
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- Publication type:
- Article
Lysosomal Acid Lipase Deficiency: Genetics, Screening, and Preclinical Study.
- Published in:
- International Journal of Molecular Sciences, 2022, v. 23, n. 24, p. 15549, doi. 10.3390/ijms232415549
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- Publication type:
- Article
Glucosylsphingosine (Lyso-Gb 1): An Informative Biomarker in the Clinical Monitoring of Patients with Gaucher Disease.
- Published in:
- International Journal of Molecular Sciences, 2022, v. 23, n. 23, p. 14938, doi. 10.3390/ijms232314938
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- Publication type:
- Article
Responses of Ileal and Fecal Microbiota to Withdrawal of Pancreatic Enzyme Replacement Therapy in a Porcine Model of Exocrine Pancreatic Insufficiency.
- Published in:
- International Journal of Molecular Sciences, 2022, v. 23, n. 19, p. 11700, doi. 10.3390/ijms231911700
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- Publication type:
- Article
Mammalian Sulfatases: Biochemistry, Disease Manifestation, and Therapy.
- Published in:
- International Journal of Molecular Sciences, 2022, v. 23, n. 15, p. 8153, doi. 10.3390/ijms23158153
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- Publication type:
- Article
Nanoemulsions as Gene Delivery in Mucopolysaccharidosis Type I—A Mini-Review.
- Published in:
- International Journal of Molecular Sciences, 2022, v. 23, n. 9, p. 4785, doi. 10.3390/ijms23094785
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- Publication type:
- Article
Comparison between Nanoparticle Encapsulation and Surface Loading for Lysosomal Enzyme Replacement Therapy.
- Published in:
- International Journal of Molecular Sciences, 2022, v. 23, n. 7, p. 4034, doi. 10.3390/ijms23074034
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- Publication type:
- Article
Understanding the Molecular Mechanisms of Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD): Towards the Development of SSADH-Targeted Medicine.
- Published in:
- International Journal of Molecular Sciences, 2022, v. 23, n. 5, p. 2606, doi. 10.3390/ijms23052606
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- Publication type:
- Article
Chaperone Therapy in Fabry Disease.
- Published in:
- International Journal of Molecular Sciences, 2022, v. 23, n. 3, p. 1887, doi. 10.3390/ijms23031887
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- Publication type:
- Article