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Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency
Adenosine deaminase (ADA) deficiency leads to severe combined immunodeficiency (SCID). Previous clinical trials showed that autologous CD34 + cell...
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Safety and tolerability of long-term treatment with darolutamide in patients with metastatic castration-resistant prostate cancer
BackgroundIn patients with metastatic castration-resistant prostate cancer, darolutamide was well tolerated for 25 months, but minimal long-term...
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Long-term safety and efficacy of open-label nabilone on sleep and pain in Parkinson´s Disease
The synthetic tetrahydrocannabinol-analog nabilone improved non-motor symptoms (NMS) in Parkinson’s disease (PD) patients in a placebo-controlled,...
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Long-term efficacy and safety of leflunomide combined with low-dose prednisone in treatment of myasthenia gravis: a retrospective study
BackgroundLeflunomide and low-dose prednisone (0.25 mg/kg/day) (LEF + Pred) rapidly improved the clinical symptoms of myasthenia gravis (MG)...
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Long-Term Constant Subcutaneous Drug Administration
In this chapter, a long-term drug delivery system for preclinical therapeutic research is introduced. By using a subcutaneously implanted ALZET®...
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Long-term effectiveness and safety of cilostazol versus clopidogrel in secondary prevention of noncardioembolic ischemic stroke
PurposeCilostazol is a widely used antiplatelet drug for secondary stroke prevention in Asia, but its comparison with clopidogrel is not well...
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Long-Term Biodistribution and Safety of Human Dystrophin Expressing Chimeric Cell Therapy After Systemic-Intraosseous Administration to Duchenne Muscular Dystrophy Model
Duchenne muscular dystrophy (DMD) is a lethal disease caused by X-linked mutations in the dystrophin gene. Dystrophin deficiency results in...
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Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome
Patients with Wiskott–Aldrich syndrome (WAS) lacking a human leukocyte antigen-matched donor may benefit from gene therapy through the provision of...
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Long-term continuous monitoring of arrhythmias in pigs with insertable cardiac monitors
Arrhythmia detection is essential when assessing the safety of novel drugs and therapies in preclinical studies. Many short-term arrhythmia...
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The IDEAL framework for surgical robotics: development, comparative evaluation and long-term monitoring
The next generation of surgical robotics is poised to disrupt healthcare systems worldwide, requiring new frameworks for evaluation. However,...
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COVID-19 and Mixed Cryoglobulinemia Syndrome: Long-Term Survey Study on the Prevalence and Outcome, Vaccine Safety, and Immunogenicity
PurposeMixed cryoglobulinemia syndrome (MCs) is a rare immunoproliferative systemic disorder with cutaneous and multiple organ involvement. Our...
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Long-term follow up of alemtuzumab-treated patients: a retrospective study in a Belgian tertiary care center
BackgroundPivotal studies have reported a significant proportion of patients achieving no evidence of disease activity (NEDA) after 2 cycles of...
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Long-term outcomes after particle radiation therapy in patients with nasopharyngeal adenoid cystic carcinoma
BackgroundNasopharyngeal adenoid cystic carcinoma (NACC) is a relatively rare salivary gland tumor that is generally associated with poor outcomes....
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The Biological Effects of Long-Term Static Magnetic Field Exposure
Although in most cases, people are exposed to static magnetic fields (SMFs) for just a short period of time, there are increasing situations where...
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Long-Term Safety and Effectiveness of PF-05280014 (a Trastuzumab Biosimilar) Treatment in Patients with HER2-Positive Metastatic Breast Cancer: Updated Results of a Randomized, Double-Blind Study
BackgroundPF-05280014 was compared with trastuzumab sourced from the European Union (trastuzumab-EU), each plus paclitaxel, as first-line treatment...
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Long-term safety and efficacy of intramyocardial adenovirus-mediated VEGF-DΔNΔC gene therapy eight-year follow-up of phase I KAT301 study
In phase I KAT301 trial, intramyocardial adenovirus-mediated vascular endothelial growth factor -D ΔNΔC (AdVEGF-D) gene therapy (GT) resulted in a...
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Early Environment and Telomeres: a Long-Term Toxic Relationship
Purpose of ReviewTelomere length (TL) shortening is a hallmark of biological aging. While studies have extensively focused on the impact of...
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Short-term and long-term outcomes of patients with gastric cancer during versus before the COVID-19 pandemic: cohort study using propensity score matching method
BackgroundThe negative effects of the novel coronavirus disease 2019 (COVID-19) pandemic on patients with gastric cancer are poorly understood. This...
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Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s disease
Glial cell line-derived neurotrophic factor (GDNF) protects dopaminergic neurons in various models of Parkinson’s disease (PD). Cell-based GDNF gene...
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A register and questionnaire study of long-term general health symptoms following SARS-CoV-2 vaccination in Denmark
Many individuals who refuse COVID-19 vaccination have concerns about long-term side effects. Here, we report findings on self-reported symptoms from...
