Advances in therapy
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Advances in therapy · May 2019
Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1.
Infants with spinal muscular atrophy (SMA) type 1 typically face a decline in motor function and a severely shortened life expectancy. Clinical trials for SMA type 1 therapies, onasemnogene abeparvovec (AVXS-101) and nusinersen, demonstrated meaningful improvements in efficacy (e.g., overall survival) but there were no head-to-head clinical trials assessing comparative efficacy. This study estimated the treatment effects of AVXS-101 relative to nusinersen for the treatment of SMA type 1. ⋯ AveXis.
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Advances in therapy · Apr 2019
Randomized Controlled TrialMatching-Adjusted Indirect Comparison of Blinatumomab vs. Inotuzumab Ozogamicin for Adults with Relapsed/Refractory Acute Lymphoblastic Leukemia.
In the absence of head-to-head trials, this analysis aimed to provide a fair indirect comparison of the efficacy between blinatumomab and inotuzumab ozogamicin (InO), two treatments for adult patients with relapsed or refractory acute lymphoblastic leukemia (R/R ALL) who received no more than one prior salvage therapy, by adjusting for cross-trial differences. ⋯ Amgen.
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Advances in therapy · Mar 2019
Randomized Controlled TrialSubgroup Analysis for Chinese Patients Included in the INPULSIS® Trials on Nintedanib in Idiopathic Pulmonary Fibrosis.
To investigate the efficacy and safety of nintedanib versus placebo in Chinese patients with idiopathic pulmonary fibrosis (IPF). ⋯ Boehringer Ingelheim. Plain language summary available for this article.
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Advances in therapy · Mar 2019
ReviewMicronized Purified Flavonoid Fraction (MPFF) for Patients Suffering from Chronic Venous Disease: A Review of New Evidence.
Chronic venous disease (CVD) is both prevalent and unavoidable in many people as a result of persistent or unalterable risk factors, the most important of which are advanced age, excess body weight, and family history. Given this inevitability, medical treatment is required to alleviate symptoms and slow disease progression. Venoactive drug therapy is emerging as a valuable treatment option for many CVD patients and micronized purified flavonoid fraction (MPFF) is the most widely prescribed and well-studied venoactive drug available. ⋯ In addition, MPFF improves the clinical signs of redness, skin changes, and edema, and improves quality of life. Collectively, these findings support the strong recommendation for MPFF treatment found in the 2018 international guidelines for the management of CVD. Funding: Servier.
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Advances in therapy · Mar 2019
Fixed-Dose Combinations of Long-Acting Bronchodilators for the Management of COPD: Global and Asian Perspectives.
Maintenance bronchodilator therapy with long-acting β-agonists (LABAs) and long-acting muscarinic antagonists (LAMAs) is the cornerstone treatment for patients with stable chronic obstructive pulmonary disease (COPD). Fixed-dose combinations (FDCs) of LABA/LAMA are recommended for the majority of symptomatic COPD patients by global guidelines; regional guidelines such as the Japanese and Korean guidelines also provide similar recommendations for the use of LABA/LAMA FDCs. This review comprehensively describes the latest clinical evidence from key studies on the efficacy and safety of four approved LABA/LAMA fixed-dose combinations: indacaterol/glycopyrronium, vilanterol/umeclidinium, formoterol/aclidinium, and olodaterol/tiotropium. ⋯ This detailed overview of the efficacy and safety of LABA/LAMA FDCs in global and Asian COPD patients is envisaged to provide a better understanding of the benefits of these therapies and to inform healthcare providers and patients on their appropriate use. Funding: Novartis Pharma K. K.