scholarly journals SUN-093 Prospective Clinical Assessment Study in Children with Achondroplasia: The PROPEL Trial

2020 ◽  
Vol 4 (Supplement_1) ◽  
Author(s):  
Ravi Savarirayan ◽  
Peter Kannu ◽  
Carl L Dambkowski ◽  
Daniela Rogoff ◽  
Melita Irving

Abstract BACKGROUND: Achondroplasia (ACH) is the most common non-lethal form of skeletal dysplasia, affecting between 1 in 15,000 to 1 in 30,000 live births [Horton et al. 2007; Waller et al. 2008]. Children and adults with ACH have disproportionate short stature, with a final height of approximately 131 cm for males and 124 cm for females. They are prone to significant co-morbidities, including obstructive sleep apnea, chronic otitis media with conductive hearing loss, spinal stenosis and a propensity towards obesity. In some infants, narrowing of the foramen magnum may result in compression of the spinal cord with neurologic sequelae, requiring timely neurosurgical intervention. There are currently no approved therapies for the treatment of ACH in either the United States or the European Union, and management is generally supportive in nature. The PROPEL trial is a prospective, non-interventional study examining baseline growth parameters and health status in children with ACH being assessed for potential enrollment into interventional studies with infigratinib, an oral FGFR1-3 inhibitor in development for achondroplasia. Methods: Children with ACH between the ages of 2.5 and 10 years are eligible for enrollment in this prospective, non-interventional trial to evaluate growth parameters and determine clinical status. Participants will be assessed at baseline, month 3, month 6, and every 6 months thereafter. The primary endpoint is annualized height growth velocity. Secondary endpoints include change from baseline in other growth parameters (including body proportionality); analysis of bone biomarkers (e.g., bone alkaline phosphatase, collagen X fragment); and the occurrence of medical events and surgical procedures. Participants will be enrolled in the study for a minimum of 6 months up to a maximum of 2 years before being considered for enrollment in a QED-sponsored phase 2/3 interventional trial. Current status: The PROPEL study is underway with the first patient enrolled in August 2019. Planned total enrollment is 200 children with ACH.

2021 ◽  
Vol 8 (1) ◽  
Author(s):  
Markku Räsänen ◽  
Henrikki Mäkynen ◽  
Mikko Möttönen ◽  
Jan Goetz

AbstractQuantum computing holds the potential to deliver great economic prosperity to the European Union (EU). However, the creation of successful business in the field is challenging owing to the required extensive investments into postdoctoral-level workforce and sophisticated infrastructure without an existing market that can financially support these operations.This commentary paper reviews the recent efforts taken in the EU to foster the quantum-computing ecosystem together with its current status. Importantly, we propose concrete actions for the EU to take to enable future growth of this field towards the desired goals. In particular, we suggest ways to enable the creation of EU-based quantum-computing unicorns which may act as key crystallization points of quantum technology and its commercialization. These unicorns may provide stability to the otherwise scattered ecosystem, thus pushing forward global policies enabling the global spread of EU innovations and technologies.The unicorns may act as a conduit, through which the EU-based quantum ecosystem can stand out from similar ecosystems based in Asia and the United States. Such strong companies are required because of the level of investment currently required in the marketplace. This paper suggests methodologies and best practices that can enhance the probability of the creation of the unicorns.Furthermore, we explore future scenarios, in which the unicorns can operate from the EU and to support the EU quantum ecosystem. This exploration is conducted focusing on the steps to be taken and on the impact the companies may have in our opinion.


2021 ◽  
Vol 0 (0) ◽  
Author(s):  
Tina L. Schuh ◽  
Leena B. Mithal ◽  
Sara Naureckas ◽  
Emily S. Miller ◽  
Craig F. Garfield ◽  
...  

Abstract Objectives We evaluated inpatient management, transition to home, breastfeeding, growth, and clinical outcomes of infants born to mothers diagnosed with SARS-CoV-2 infection in pregnancy and followed in a Federally Qualified Health Center (FQHC), that serves a diverse and low-income patient population, from birth through 6 months of life. Methods Infants born between 4/3/20 and 7/26/20 at Prentice Women’s Hospital with mothers who received prenatal care at Erie Family Health Center (Erie), the second largest FQHC in Illinois, and had confirmed SARS-CoV-2 during pregnancy were included. Data were abstracted from delivery hospital admission and outpatient follow-up appointments between 4/8/20 and 2/4/21. Results Thirty-three infants met inclusion criteria. Average gestational age was 38.9 weeks (IQR 37.6–40.4), 3 (10%) were premature and 5 (15%) required NICU admission. Nearly all (97%) mothers expressed intent to breastfeed. Outpatient follow-up rates were similar to historical cohorts and 82% (23/28) of infants were vaccination compliant. Growth parameters showed normal distributions at all time points. At 6 months, any and exclusive breast milk feeding rates were lower compared to historic cohorts (18 vs. 36%, p<0.05, 0 vs. 21%, p<0.01). Three infants (10%) received development-related referrals, one carried an underlying genetic diagnosis. Outpatient visits were predominantly face-to-face with telemedicine use comprising only 6% of visits (11/182). Conclusions Longitudinal follow-up of 33 publicly insured infants born to mothers with SARS-CoV-2 infection in pregnancy followed in an FQHC showed high rates of follow-up and vaccination compliance, normal growth patterns and reassuring clinical status, and lower than expected rates of breastfeeding.


2018 ◽  
Vol 2018 (6) ◽  
pp. 3-12
Author(s):  
Zhang DONGYANG ◽  

The status and prospects of development of trade and economic relations between Ukraine and China are considered. It is proved that bilateral cooperation in the trade and economic sphere has made significant progress. In 2012–2017, China was the second largest trading partner of Ukraine after Russia. However, the problem of imbalance in imports and exports between Ukraine and China has not yet been resolved. In addition, the scale and number of projects in which Ukraine attracts Chinese investment is much less than investments from European countries and the United States. It is justified that trade and economic cooperation between Ukraine and China is at a new historical stage. On the one hand, Ukraine signed the Association Agreement with the European Union, and on January 1, 2016, the rules of the free trade zone between Ukraine and the EU entered into force. This helps to accelerate the integration of Ukrainian economy into European one. On the other hand, the global economic downturn requires the introduction of innovations in the model of cooperation. The Chinese initiative “One belt is one way” is one of the variants of the innovation model of cooperation. Its significance is to unite the Asia-Pacific region with the EU in order to join the Eurasian Economic Union, create a new space and opportunities for development and achieve prosperity with the Eurasian countries. All this forms unprecedented opportunities for development of bilateral economic and trade relations. It seems that to fully open the potential of Ukrainian economy and expand bilateral trade and economic cooperation, it is necessary to take into account such proposals as the establishment of the Sino-Ukrainian industrial park, the promotion of cooperation in the field of electronic commerce, the formation of the Sino-Ukrainian free trade zone and enhanced interaction within multilateral mechanisms (for example, the Shanghai Cooperation Organization and the interaction of China and the countries of Central and Eastern Europe in the 16 + 1 format).


Author(s):  
Richard Pomfret

This book analyzes the Central Asian economies of Kazakhstan, the Kyrgyz Republic, Tajikistan, Turkmenistan, and Uzbekistan, from their buffeting by the commodity boom of the early 2000s to its collapse in 2014. The book examines the countries' relations with external powers and the possibilities for development offered by infrastructure projects as well as rail links between China and Europe. The transition of these nations from centrally planned to market-based economic systems was essentially complete by the early 2000s, when the region experienced a massive increase in world prices for energy and mineral exports. This raised incomes in the main oil and gas exporters, Kazakhstan and Turkmenistan; brought more benefits to the most populous country, Uzbekistan; and left the poorest countries, the Kyrgyz Republic and Tajikistan, dependent on remittances from migrant workers in oil-rich Russia and Kazakhstan. The book considers the enhanced role of the Central Asian nations in the global economy and their varied ties to China, the European Union, Russia, and the United States. With improved infrastructure and connectivity between China and Europe (reflected in regular rail freight services since 2011 and China's announcement of its Belt and Road Initiative in 2013), relaxation of UN sanctions against Iran in 2016, and the change in Uzbekistan's presidency in late 2016, a window of opportunity appears to have opened for Central Asian countries to achieve more sustainable economic futures.


Author(s):  
Attarid Awadh Abdulhameed

Ukrainia Remains of huge importance to Russian Strategy because of its Strategic importance. For being a privileged Postion in new Eurasia, without its existence there would be no logical resons for eastward Expansion by European Powers.  As well as in Connection with the progress of Ukrainian is no less important for the USA (VSD, NDI, CIA, or pentagon) and the European Union with all organs, and this is announced by John Kerry. There has always ben Russian Fear and Fear of any move by NATO or USA in the area that it poses a threat to  Russians national Security and its independent role and in funence  on its forces especially the Navy Forces. There for, the Crisis manyement was not Zero sum game, there are gains and offset losses, but Russia does not accept this and want a Zero Sun game because the USA. And European exteance is a Foot hold in Regin Which Russian sees as a threat to its national security and want to monopolize control in the strategic Qirim.


2019 ◽  
Vol 02 (03) ◽  
Author(s):  
Sherif Aly ◽  
Allan Stolarski ◽  
Patrick O’Neal ◽  
Edward Whang ◽  
Gentian Kristo

2019 ◽  
Vol 22 (2) ◽  
pp. 74-79
Author(s):  
Nargiza Sodikova ◽  
◽  
◽  

Important aspects of French foreign policy and national interests in the modern time,France's position in international security and the specifics of foreign affairs with the United States and the European Union are revealed in this article


2016 ◽  
pp. 26-46
Author(s):  
Marcin Jan Flotyński

The global financial crisis in 2007–2009 began a period of high volatility on the financial markets. Specifically, it caused an increased amplitude of fluctuations of the level of gross domestic products, the level of investment and consumption and exchange rates in particular countries. To address the adverse market circumstances, governments and central banks took actions in order to bolster the weakening global economy. The aim of this article is to present the anti-crisis actions in the United States and selected member states of the European Union, including Poland, and an assessment of their efficiency. The analysis conducted indicates that generally the actions taken in the United States in response to the crisis were faster and more adequate to the existing circumstances than in the European Union.


2019 ◽  
Vol 17 (3) ◽  
pp. 65-77
Author(s):  
Martin Dahl

When the political camp centred on the Law and Justice party (PiS) came to power in 2015, it led to a change in priorities in Polish foreign policy. The Three Seas Initiative (TSI), understood as closer cooperation between eastern states of the European Union in the area between the Baltic, Adriatic, and Black seas, has become a new instrument of foreign policy. The initiative demonstrates the growing importance of Central and Eastern Europe in the global game of great powers. The region has become a subject of rivalry, not only between the United States and Russia but also China. Therefore, the main objective of this article is to try to describe the importance of the region to Germany and how Germany’s stance on the TSI has evolved. The article consists of three parts, an introduction to the issues, the genesis of the TSI, and the definition of goals set by the states participating in this initiative, as well as analysis of the German stance towards the initiative since its development in 2015. The theories of geopolitics and neorealism are used as the theoretical basis for the analysis.


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