Transthyretin (ATTR) amyloidosis is a multisystem disease caused by organ deposition of amyloid fibrils derived from the misfolded transthyretin (TTR) protein. The purpose of this article is to provide an overview of current treatment regimens and summarize important considerations for each agent. A literature search was performed with the PubMed database for articles published through October 2020. Search criteria included therapies available on the market and investigational therapies used for ATTR amyloidosis treatment. Both prospective clinical trials and retrospective studies have been included in this review. Available therapies discussed in this review article are tafamidis, diflunisal, patisiran, and inotersen. Tafamidis is FDA approved for treatment of wild-type ATTR (ATTRwt) and hereditary ATTR (ATTRv) cardiomyopathy, and patisiran and inotersen are FDA approved for ATTRv polyneuropathy. Diflunisal does not have an FDA-labeled indication for amyloidosis but has been studied in ATTRv polyneuropathy and ATTRwt cardiomyopathy. Investigational therapies include a TTR stabilizer, AG10; 2 antifibril agents, PRX004 and doxycycline/ tauroursodeoxycholic acid; and 2 gene silencers, vutrisiran and AKCEA-TTR-LRx; and clinical trials are ongoing. ATTR amyloidosis treatment selection is based on subtype and presence of cardiac or neurological manifestations. Additional considerations such as side effects, monitoring, and administration are outlined in this review.
This article examines the 2013 migration policy liberalizations in Morocco and Turkey in order to understand whether predominantly “human rights-centric” or “diplomatic” factors influenced domestic decisions to reform migration policies. It uses original interview data collected in 2015, as well as policy documents, to examine the two reform processes and their initial consequences for migrants and refugees residing in each host state. While the academic literature on migration has focused on human rights-centric factors to understand historic migration policy reforms, Turkey and Morocco’s geopolitical and geographic positions between powerful neighbors to the north and important sending countries to the south mean that diplomatic factors are also key to understanding the incentives behind reform. This article’s findings have important implications for scholars of international migration, demonstrating that while countries like Morocco and Turkey may implement liberal and inclusive policies if there are diplomatic and economic gains to be had from doing so, such policies may have little impact on the everyday lives of individual migrants and refugees residing in these states and may be subject to reversals if such states’ geopolitical calculations change.
Since the 2015 refugee “crisis,” much has been made of the distinction between the legal category of refugee and migrant. While migration scholars have accounted for the increased blurring of these two categories through explanations of institutional drift and policy layering, we argue that the intentional policies utilized by states and international organizations to minimize legal avenues for refugees to seek protection should also be considered. We identify four practices of policy “conversion” that have also led to the increasingly problematic distinction between migrants and refugees: (1) limiting access to territory through burden-shifting and other practices of extraterritorialization; (2) limiting access to asylum and local integration through procedural and administrative hindrances; (3) the use of group-based criteria as a basis of exclusion; (4) the inclusion of non-Convention criteria within resettlement schemes. Drawing upon a historical institutionalist approach and a wide array of empirical sources—including 3 years of combined primary field research conducted in Egypt, Lebanon, Morocco, Tunisia, and Turkey between 2013 and 2016—we demonstrate that states are actively pursuing a greater degree of control over the selection of refugees, in practice making refugee resettlement closer to another immigration track rather than a unique status that compels state responsibility.
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