Results 91 to 100 of about 6,701,012 (264)
Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy
Pediatric Pulmonology, 2018 Spinal Muscular Atrophy type 1 (SMA1) is a rare genetic neuromuscular disease where 75% of SMA1 patients die/require permanent‐ventilation by 13.6 months.
S. Al-Zaidy +15 more
semanticscholar +1 more source
Enzyme replacement therapy: efficacy and limitations
Italian Journal of Pediatrics, 2018 Enzyme replacement therapy (ERT) is available for mucopolysaccharidosis (MPS) I, MPS II, MPS VI, and MPS IVA. The efficacy of ERT has been evaluated in clinical trials and in many post-marketing studies with a long-term follow-up for MPS I, MPS II, and ...
D. Concolino, F. Deodato, R. Parini
semanticscholar +1 more source
Reality Versus Grant Application Research “Plans” [PDF]
, 2018 This article describes the implementation of the American Indian mHealth Smoking Dependence Study focusing on the differences between what was written in the grant application compared to what happened in reality.
Addo, Ivor D. +13 more
core +1 more source
Metachromatic Leukodystrophy: Diagnosis, Modeling, and Treatment Approaches
Frontiers in Medicine, 2020 Metachromatic leukodystrophy is a lysosomal storage disease, which is characterized by damage of the myelin sheath that covers most of nerve fibers of the central and peripheral nervous systems.
Alisa A. Shaimardanova +8 more
doaj +1 more source
Subcutaneous IgG Replacement Therapy by Push in 32 Patients with Primary Immunodeficiency Diseases in Argentine [PDF]
, 2014 Introduction: Regular replacement with immunoglobulin infusions is the mainstay of treatment in the majority of primary immunodeficiencies. Several studies showed that Subcutaneous Immunoglobulin (SCIG) has similar efficacy to Intravenous Immunoglobulin (
Bezrodnik, Liliana +6 more
core +1 more source
Surfactant replacement therapy
Jornal de Pediatria, 2001 OBJECTIVE: To analyze and update information about surfactant therapy replacement in newborns with lung diseases. SOURCES: Literature review, including textbooks, meta-analyses, prospective, randomized controlled trials, retrospective assessments and case studies. Literature was reviewed based on the authors clinical and scientific experience regarding
openaire +2 more sources
Renal Replacement TherapyBackground Japanese peritoneal dialysis (PD) guidelines do not suggest applying mupirocin/gentamicin ointment to the exit sites of PD patients to prevent exit-site infection (ESI).
Nao Asai +14 more
doaj +1 more source
Hyperuricemia and prognosis in chronic kidney disease
Počki, 2017 The ratio of uricemia, magnesia, quality of life and functional reserve of the kidneys in CKD has not been completely determined yet. These rates were analysed in 145 patients, depending on the CKD 1–5 stages.
D.D. Ivanov +2 more
doaj +1 more source
Intradermally delivered mRNA-encapsulating extracellular vesicles for collagen-replacement therapy
Nature Biomedical Engineering, 2023 Yi You +29 more
semanticscholar +1 more source
INTRAVENOUS IMMUNOGLOBULIN APPLICATION IN CLINICAL PRACTICE
Педиатрическая фармакология, 2013 The article gives data on the structure and function of immunoglobulins. Given the multitude of mechanisms of their action, indications to their use are constantly expanding.
T. V. Latysheva, E. A. Latysheva
doaj +1 more source