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Novartis ’ Spinal Drug Gets FDA Approval, $2 Million Price Tag
(CNN) — The US Food and Drug Administration approved a treatment Friday for a genetic disease called spinal muscular atrophy that causes infants’ muscles to waste away, potentially killing them before age 2. And then came the price tag: $2.125 million for a one-time treatment. The gene therapy, called Zolgensma, will be marketed by AveXis, whose parent company is Novartis. “Today’s approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases,” Dr. Ned Sharpless, the FDA’s acting commissioner, said in a statement Friday. ̶...
Source: WBZ-TV - Breaking News, Weather and Sports for Boston, Worcester and New Hampshire - May 24, 2019 Category: Consumer Health News Authors: Health – CBS Boston Tags: Health News CNN Novartis Source Type: news

Sickle Cell Disease Subjects Have a Distinct Abnormal Autonomic Phenotype Characterized by Peripheral Vasoconstriction With Blunted Cardiac Response to Head-Up Tilt
Conclusion We have shown that SCD subjects are much more likely than non-SCD subjects to have impaired cardiac, but intact peripheral responses to orthostatic stress induced by HUT. These abnormal responses are associated with low baseline cardiac parasympathetic activity, independent of hemoglobin level. The classification of autonomic phenotypes based on HUT response may have potential use for predicting disease severity, guiding and targeting treatments/interventions to alleviate the risk of adverse outcomes in SCD. Ethics Statement All experiments were conducted at Children’s Hospital Los Angeles (CHLA). The ...
Source: Frontiers in Physiology - April 10, 2019 Category: Physiology Source Type: research

Urine Arsenic and Arsenic Metabolites in U.S. Adults and Biomarkers of Inflammation, Oxidative Stress, and Endothelial Dysfunction: A Cross-Sectional Study
Conclusion: In a cross-sectional study of U.S. adults, we observed some positive associations of uAs and toenail As concentrations with biomarkers potentially relevant to CVD pathogenesis and inflammation, and evidence of a higher capacity to metabolize inorganic As was negatively associated with a marker of oxidative stress. https://doi.org/10.1289/EHP2062 Received: 14 April 2017 Revised: 13 November 2017 Accepted: 15 November 2017 Published: 15 December 2017 Address correspondence to S.F. Farzan, 2001 N. Soto St., Los Angeles, CA, 90032. Telephone: (323)-442-5101; Email: sffarzan@usc.edu Supplemental Material is ava...
Source: EHP Research - December 16, 2017 Category: Environmental Health Authors: Daniil Lyalko Tags: Research Source Type: research