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. “The potential for gene therapy products to change the lives of those patients who may have faced a terminal condition, or worse, death, provides hope for the future.” The list price, which was anticipated to be in the low millions, immediately reignited controversy around the rising costs of drugs and how to pay for them. “It is emblematic of our broken system that effectively forces us to pay whatever price drug corporations demand for lifesaving new drugs,” David Mitchell, founder of the advocacy group Patients For Affordable Drugs, said in a statement Friday. Novartis leaders said Friday that the price met their targets of cost-effectiveness, with the newly approved treatment costing about half of what another treatment for the disorder, Spinraza, might cost over 10 years. They said that the company will have patient support and paymen...
Source: WBZ-TV - Breaking News, Weather and Sports for Boston, Worcester and New Hampshire - Category: Consumer Health News Authors: Tags: Health News CNN Novartis Source Type: news

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Abstract In this work, the as-prepared V2O5 nanobelts can sensitively quench the fluorescence of nitrogen-doped carbon dots (N-CDs) based on the inner filter effect (IFE). In the presence of ascorbic acid (AA), the fluorescence of N-CDs can recover through the redox reaction between V2O5 nanobelts and AA. Meanwhile, in the presence of both alkaline phosphatase (ALP) and ascorbyl-2-phosphate (AAP), the fluorescence of N-CDs can also restore since AAP can be hydrolyzed into AA by ALP. Under optimum conditions, the linear range for AA is from 0.01 to 2.5 μM with a detection limit of 3 nM and that for ALP is fr...
Source: Analytica Chimica Acta - Category: Chemistry Authors: Tags: Anal Chim Acta Source Type: research
Chem. Commun., 2019, Accepted Manuscript DOI: 10.1039/C9CC07253E, CommunicationXuehan Yin, Ting Hou, Bingzheng Huang, Limin Yang, Feng Li We developed an aptamer recognition-trigged label-free homogeneous electrochemical biosensing method for highly selective and ultrasensitive detection of cancer-derived exosomes. The content of this RSS Feed (c) The Royal Society of Chemistry
Source: RSC - Chem. Commun. latest articles - Category: Chemistry Authors: Source Type: research
ConclusionsPhysical and psychological functioning, pain, fatigue and appetite loss had prognostic significance above and beyond clinical predictors in CRC. Routine monitoring of these PROs may allow earlier detection and amelioration of problems, which may improve quality of life and perhaps extend survival. More research is needed to determine prognostic value of PROs in early-stage CRC, and prognostic significance of changes in PRO scores.
Source: Quality of Life Research - Category: Health Management Source Type: research
This study aims to address this gap.MethodsThe study used data from the Nihon University Japanese Longitudinal Study of Aging (NUJLSOA) and incidence-based multistate life tables to estimate disabled and disability-free life expectancy based on the stroke status of Japanese residents aged 65 and older.ResultsJapanese stroke survivors aged 65 who experienced an initial disability-free state could expect to live approximately 3 fewer total years of life, 4 –5 fewer years in a disability-free state, and 1–2 more years in a disabled state compared to those without history of a stroke (p 
Source: Quality of Life Research - Category: Health Management Source Type: research
ConclusionsSelf-rated health of the general US adult population in 2017 was similar to 2002, but after adjusting for health problems, scores were slightly higher in 2017.  Sociodemographic shifts in age and education explain some of the differences in scores, and by removing health and sociodemographic factors, we found the VAS reveals self-rated health is slightly better in 2017 than 2002.
Source: Quality of Life Research - Category: Health Management Source Type: research
ConclusionsThe experience of changes in taste was common yet highly variable in its presentation and resultant changes in food preferences. Taste changes did not always resolve soon after treatment completion. Future research should identify ways to manage this symptom in pediatric cancer patients.
Source: Quality of Life Research - Category: Health Management Source Type: research
BACKGROUND: The meniscus is vital for load bearing, knee stabilization, and shock absorption, making a meniscal tear a well-recognized sport-related injury in children and young adults. An inverse relationship between the quality and value of orthopaedic c...
Source: SafetyLit - Category: International Medicine & Public Health Tags: Age: Adolescents Source Type: news
Over the last 10 years, the rate of adolescents ages 15 to 19 committing suicide in the United States increased drastically, with an overall rate of 6.7 suicides per 100  000 per year in 2007 and a rate of 11.8 per 100 000 per year in 2017.1 The rat...
Source: SafetyLit - Category: International Medicine & Public Health Tags: Age: Adolescents Source Type: news
BACKGROUND: High school athletes with lower extremity sports injuries (LESIs) frequently present to the emergency department in the United States. Previous epidemiological studies have presented rates and mechanisms of LESIs in these athletes. No studies, ...
Source: SafetyLit - Category: International Medicine & Public Health Tags: Age: Adolescents Source Type: news
OBJECTIVE: We conducted a scoping review of the literature to assess the prevalence of hospital policies regarding emergency contraception (EC) and the frequency that U.S. emergency departments (EDs) provide EC to sexual assault survivors. STUDY DESIGN: We...
Source: SafetyLit - Category: International Medicine & Public Health Tags: Age: Adolescents Source Type: news
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