scholarly journals Onasemnogene Abeparvovec-xioi: World’s most expensive and approved therapy for spinal muscular atrophy

2021 ◽  
pp. 145-149
Author(s):  
Vinod Bairagi
Keyword(s):  
United States ◽  
Gene Therapy ◽  
Spinal Muscular Atrophy ◽  
Healthcare Professionals ◽  
Muscular Atrophy ◽  
The United States ◽  
Severe Form ◽  
Expensive Drug ◽  
The U.S ◽  
Experience Difficulty

On May 24, 2019, the U.S. Food and Drug Administration approved Onasemnogene Abeparvovec-xioi (Zolgensma), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe form of SMA and a leading genetic cause of infant mortality. It is priced in the United States at $2.1m (£1.6m; €1.9m) the world’s most expensive drug. Children with SMA experience difficulty performing essential functions of life. Most children with this disease do not survive past early childhood due to respiratory failure. Patients with SMA now have another treatment option to minimize the progression of SMA and improve survival. This review summarized the clinical guidelines for the Onasemnogene Abeparvovec-xioi use and may be useful to healthcare professionals.

scholarly journals Economic burden of spinal muscular atrophy in the United States: a contemporary assessment

2019 ◽  
Vol 23 (1) ◽  
pp. 70-79 ◽  
Author(s):  
Marcus Droege ◽  
Douglas Sproule ◽  
Ramesh Arjunji ◽  
Marjolaine Gauthier-Loiselle ◽  
Martin Cloutier ◽  
...  
Keyword(s):  
United States ◽  
Spinal Muscular Atrophy ◽  
Economic Burden ◽  
Muscular Atrophy ◽  
The United States

scholarly journals Landscape of Spinal Muscular Atrophy Newborn Screening in the United States: 2018–2021

2021 ◽  
Vol 7 (3) ◽  
pp. 33
Author(s):  
Kshea Hale ◽  
Jelili Ojodu ◽  
Sikha Singh
Keyword(s):  
United States ◽  
Spinal Muscular Atrophy ◽  
Newborn Screening ◽  
Human Services ◽  
Muscular Atrophy ◽  
Genetic Disorders ◽  
The United States ◽  
Life Saving ◽  
Increased Risk ◽  
Review Current

Newborn screening (NBS) programs identify newborns at increased risk for genetic disorders, linking these newborns to timely intervention and potentially life-saving treatment. In the United States, the Health and Human Services (HHS) Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) recommends the disorders for state NBS programs to screen. ACHDNC updated the Recommended Uniform Screening Panel to include Spinal Muscular Atrophy (SMA) in July 2018. As of June 2021, 34 state NBS programs had fully implemented SMA newborn screening, and at least 8 programs were pursuing implementation. This article will review current SMA screening processes, considerations, challenges, and status.

scholarly journals Nusinersen for Spinal Muscular Atrophy in the United States: Findings From a Retrospective Claims Database Analysis

2021 ◽  
Author(s):  
Marjolaine Gauthier-Loiselle ◽  
Martin Cloutier ◽  
Walter Toro ◽  
Anish Patel ◽  
Sherry Shi ◽  
...  
Keyword(s):  
United States ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
The United States ◽  
Database Analysis ◽  
Claims Database

An American Language

2018 ◽  
Author(s):  
Rosina Lozano
Keyword(s):  
United States ◽  
National Policy ◽  
The United States ◽  
Spanish Language ◽  
Spanish Speakers ◽  
Language Recognition ◽  
Political Language ◽  
Political Issue ◽  
History Of ◽  
The U.S

An American Language is a political history of the Spanish language in the United States. The nation has always been multilingual and the Spanish language in particular has remained as an important political issue into the present. After the U.S.-Mexican War, the Spanish language became a language of politics as Spanish speakers in the U.S. Southwest used it to build territorial and state governments. In the twentieth century, Spanish became a political language where speakers and those opposed to its use clashed over what Spanish's presence in the United States meant. This book recovers this story by using evidence that includes Spanish language newspapers, letters, state and territorial session laws, and federal archives to profile the struggle and resilience of Spanish speakers who advocated for their language rights as U.S. citizens. Comparing Spanish as a language of politics and as a political language across the Southwest and noncontiguous territories provides an opportunity to measure shifts in allegiance to the nation and exposes differing forms of nationalism. Language concessions and continued use of Spanish is a measure of power. Official language recognition by federal or state officials validates Spanish speakers' claims to US citizenship. The long history of policies relating to language in the United States provides a way to measure how U.S. visions of itself have shifted due to continuous migration from Latin America. Spanish-speaking U.S. citizens are crucial arbiters of Spanish language politics and their successes have broader implications on national policy and our understanding of Americans.

Palestine Unbound

2018 ◽  
Vol 47 (3) ◽  
pp. 130-134
Keyword(s):  
United States ◽  
Human Rights ◽  
Task Force ◽  
The United States ◽  
Donald Trump ◽  
Tel Aviv ◽  
The Us ◽  
Intention To Move ◽  
Arab Israeli Conflict ◽  
The U.S

This section, updated regularly on the blog Palestine Square, covers popular conversations related to the Palestinians and the Arab-Israeli conflict during the quarter 16 November 2017 to 15 February 2018: #JerusalemIstheCapitalofPalestine went viral after U.S. president Donald Trump recognized Jerusalem as the capital of Israel and announced his intention to move the U.S. embassy there from Tel Aviv. The arrest of Palestinian teenager Ahed Tamimi for slapping an Israeli soldier also prompted a viral campaign under the hashtag #FreeAhed. A smaller campaign protested the exclusion of Palestinian human rights from the agenda of the annual Creating Change conference organized by the US-based National LGBTQ Task Force in Washington. And, UNRWA publicized its emergency funding appeal, following the decision of the United States to slash funding to the organization, with the hashtag #DignityIsPriceless.

The French Way

2011 ◽  
Author(s):  
Richard F. Kuisel
Keyword(s):  
United States ◽  
The United States ◽  
French Language ◽  
French Cinema ◽  
Free Markets ◽  
Technology Sector ◽  
Aids Epidemic ◽  
Political Reality ◽  
Politics And Culture ◽  
The U.S

There are over 1,000 McDonald's on French soil. Two Disney theme parks have opened near Paris in the last two decades. And American-inspired vocabulary such as “le weekend” has been absorbed into the French language. But as former French president Jacques Chirac put it: “The U.S. finds France unbearably pretentious. And we find the U.S. unbearably hegemonic.” Are the French fascinated or threatened by America? They Americanize yet are notorious for expressions of anti-Americanism. From McDonald's and Coca-Cola to free markets and foreign policy, this book looks closely at the conflicts and contradictions of France's relationship to American politics and culture. The book shows how the French have used America as both yardstick and foil to measure their own distinct national identity. France has charted its own path: it has welcomed America's products but rejected American policies; assailed Americ's “jungle capitalism” while liberalizing its own economy; attacked “Reaganomics” while defending French social security; and protected French cinema, television, food, and language even while ingesting American pop culture. The book examines France's role as an independent ally of the United States, but he also considers the country's failures in influencing the Reagan, Bush, and Clinton administrations. Whether investigating France's successful information technology sector or its spurning of American expertise during the AIDS epidemic, the book asks if this insistence on a French way represents a growing distance between Europe and the United States or a reaction to American globalization. Exploring cultural trends, values, public opinion, and political reality, this book delves into the complex relationship between two modern nations.

Latino Catholicism

2011 ◽  
Author(s):  
Timothy Matovina
Keyword(s):  
United States ◽  
Sixteenth Century ◽  
Catholic Church ◽  
Social Life ◽  
Social Issues ◽  
The United States ◽  
Societal Impact ◽  
Comprehensive Overview ◽  
Public Debates ◽  
The U.S

Most histories of Catholicism in the United States focus on the experience of Euro-American Catholics, whose views on social issues have dominated public debates. This book provides a comprehensive overview of the Latino Catholic experience in America from the sixteenth century to today, and offers the most in-depth examination to date of the important ways the U.S. Catholic Church, its evolving Latino majority, and American culture are mutually transforming one another. This book highlights the vital contributions of Latinos to American religious and social life, demonstrating in particular how their engagement with the U.S. cultural milieu is the most significant factor behind their ecclesial and societal impact.

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