Regulating Rare Disease: Safely Facilitating Access to Orphan Drugs

While approximately one in ten Americans suffers from a rare disease, only 5 percent of rare diseases have a U.S. Food and Drug Administration (FDA) approved treatment. Congressional and regulatory efforts to stimulate the development of rare-disease treatments, while laudable, have not resolved the fundamental issues surrounding rare-disease treatment development. Indeed, small patient populations, incomplete scientific understanding of rare diseases, and high development costs continually limit the availability of rare-disease treatments. To illustrate the struggle of developing and approving safe rare-disease treatments, this Note begins by discussing the approval of Eteplirsen, the first drug approved for treating a rare disease called Duchenne muscular dystrophy. After exploring the current drug regulation system and how this impacts the availability of rare-disease treatments, this Note examines the 21st Century Cures Act’s patient experience data provisions and the currently pending Trickett Wendler Right to Try Act. Ultimately, the unmet therapeutic needs of rare-disease patients can be met while protecting patient safety, this Note reasons that, if carefully implemented, the 21st Century Cures Act and the Trickett Wendler Right to Try Act could work in tandem to safely facilitate patient access to rare-disease treatments

A microscopic study of solid/liquid phase change in several members of the paraffin family

Microscopic study of solid/liquid phase change in several members of paraffin famil

Missional Apologetics: An Examination of Essential Elements in the Apologetic Approaches of Early Christian Era Apologists in Light of the Mission of Christ to a Pluralistic World

The cultural ethos known as postmodernism has impacted the epistemological and functional structures of contemporary society in dramatic ways. Changes in the attitudes, standards, and belief systems from which people operate in their day-to-day lives have seen radical alterations in the last fifty years. The impact of the postmodern milieu has been evident in various ways in local church ministry, as well as the extension of local church ministry on the mission field.As a result of the waning effectiveness of traditional evangelism methods and indications that there is a relationship between that waning efficacy and the postmodern milieu, scholars have attempted to formulate strategies for effectively penetrating the culture with the gospel. There is an urgent need for pre-evangelism tools aimed at garnering the thoughtful consideration of the Christian message by a society identified as postmodern, post-evangelical, and post-Christian by researchers and scholars. Apologetics has historically filled the needed role in presenting arguments and evidence designed to legitimize the Christian worldview in cultures where it was not seen as a viable option. This work proposes that a Missional Apologetic approach is also a viable option for promoting the Christian Worldview in the post-Christian world. The Missional Apologetic approach proposed must find its basis in the Mission of Christ (Missio Christi) as defined in the Gospels and Acts and as extended in the works of the Early Church apologists. The similarities of the culture versus Christian conflict in the early Christian era and post-Christian era cultures, makes it is necessary to take a deeper look at the apologists who defended the Christian faith in the pre-Christian culture. Taking a closer look at the apologies of men like Aristides, Justin Martyr, Tatian, and Tertullian can provide some principles that take the work of apologetics in the post-Christian culture to a higher level of effectiveness

Consequences of a Distant Massive Planet on the Large Semi-major Axis Trans-Neptunian Objects

We explore the distant giant planet hypothesis by integrating the large semi-major axis, large pericenter Trans-Neptunian Objects (TNOs) in the presence of the giant planets and an external perturber whose orbit is consistent with the proposed distant, eccentric, and inclined giant planet, so called planet 9. We find that TNOs with semi-major axes greater than 250 au experience some longitude of perihelion shepherding, but that a generic outcome of such evolutions is that the TNOs evolve to larger pericenter orbits, and commonly get raised to retrograde inclinations. This pericenter and inclination evolution requires a massive disk of TNOs (tens of M_\Earth) in order to explain the detection of the known sample today. Some of the highly inclined orbits produced by the examined perturbers will be inside of the orbital parameter space probed by prior surveys, implying a missing signature of the 9th planet scenario. The distant giant planet scenarios explored in this work do not reproduce the observed signal of simultaneous clustering in argument of pericenter, longitude of the ascending node, and longitude of perihelion in the region of the known TNOs

Fast track children's hearing pilot: final report of the evaluation of the pilot

This report presents key findings of the evaluation of the Fast Track children’s hearings pilot in Scotland1. The research was undertaken by staff at the Universities of Glasgow, Stirling and Strathclyde between February 2003 and January 2005

Oxygen Isotopes of Al-Rich Chondrules from Unequilibrated Ordinary Chondrites

Al-rich chondrules (ARCs) are a rare constituent of chondrites. They have relatively high bulk Al_2O_3 content (> 10 wt%), which is due to the presence of Al-rich phases, such as plagioclase, spinel, Al-rich glass etc. [1]. ARCs share some chemical and petrologic characteristics with Ca, Al-rich inclusions (CAis), and may represent a genetic link between ferromagnesian chondrules and CAis

Ensemble prediction for nowcasting with a convection-permitting model - II: forecast error statistics

A 24-member ensemble of 1-h high-resolution forecasts over the Southern United Kingdom is used to study short-range forecast error statistics. The initial conditions are found from perturbations from an ensemble transform Kalman filter. Forecasts from this system are assumed to lie within the bounds of forecast error of an operational forecast system. Although noisy, this system is capable of producing physically reasonable statistics which are analysed and compared to statistics implied from a variational assimilation system. The variances for temperature errors for instance show structures that reflect convective activity. Some variables, notably potential temperature and specific humidity perturbations, have autocorrelation functions that deviate from 3-D isotropy at the convective-scale (horizontal scales less than 10 km). Other variables, notably the velocity potential for horizontal divergence perturbations, maintain 3-D isotropy at all scales. Geostrophic and hydrostatic balances are studied by examining correlations between terms in the divergence and vertical momentum equations respectively. Both balances are found to decay as the horizontal scale decreases. It is estimated that geostrophic balance becomes less important at scales smaller than 75 km, and hydrostatic balance becomes less important at scales smaller than 35 km, although more work is required to validate these findings. The implications of these results for high-resolution data assimilation are discussed