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1.
Cells ; 13(10)2024 May 16.
Artigo em Inglês | MEDLINE | ID: mdl-38786070

RESUMO

Sickle cell disease (SCD) is the most common genetic blood disorder in the United States, with over 100,000 people suffering from this debilitating disease. SCD is caused by abnormal hemoglobin (Hb) variants that interfere with normal red blood cell (RBC) function. Research on SCD has led to the development and approval of several new SCD therapies in recent years. The recent FDA-approved novel gene therapies are potentially curative, giving patients an additional option besides a hematopoietic bone marrow transplant. Despite the promise of existing therapies, questions remain regarding their long-term pharmacological effects on adults and children. These questions, along with the exorbitant cost of the new gene therapies, justify additional research into more effective therapeutic options. Continual research in this field focuses on not only developing cheaper, more effective cures/treatments but also investigating the physiological effects of the current therapies on SCD patients, particularly on the brain and kidneys. In this article, we undertake a comprehensive review of ongoing clinical trials with completion dates in 2024 or later. Our exploration provides insights into the landscape of current therapeutics and emerging novel therapies designed to combat and potentially eradicate SCD, including the latest FDA-approved gene therapies.


Assuntos
Anemia Falciforme , Terapia Genética , Humanos , Anemia Falciforme/terapia , Anemia Falciforme/genética , Ensaios Clínicos como Assunto
2.
J Pediatr (Rio J) ; 89(1): 100-7, 2013.
Artigo em Inglês | MEDLINE | ID: mdl-23544817

RESUMO

OBJECTIVE: This study investigated the association between alcohol and violence among Argentine youth. METHODS: Data from the 2007 Argentina Global School-based Student Health Survey (GSHS), a nationally representative survey of middle school students, were examined using age-adjusted logistic regression models. RESULTS: Of the 1,328 participating students aged 13 to 15 years old, 51.9% reported drinking alcohol in the previous month, with higher rates among males (p=0.04) and older students (p<0.01). Both male and female drinkers were nearly twice as likely as non-drinkers to report being physically attacked, being in a physical fight, and having thoughts about self-directed violence. Among drinkers, those who reported poor mental health, were victims of bullying, used tobacco or drugs, or skipped school without permission were approximately twice as likely as other drinkers to have engaged in violent activities. CONCLUSION: Public health interventions targeting violence among young adolescents should be developed in combination with alcohol education programs.


Assuntos
Comportamento do Adolescente/psicologia , Consumo de Bebidas Alcoólicas/epidemiologia , Vigilância da População/métodos , Violência/estatística & dados numéricos , Adolescente , Consumo de Bebidas Alcoólicas/psicologia , Argentina/epidemiologia , Bullying , Estudos Transversais , Feminino , Humanos , Masculino , Saúde Mental/estatística & dados numéricos , Prevalência , Comportamento Autodestrutivo/complicações , Distribuição por Sexo , Fatores Sexuais , Violência/psicologia
3.
J. pediatr. (Rio J.) ; 89(1): 100-107, jan.-fev. 2013. tab
Artigo em Português | LILACS | ID: lil-668832

RESUMO

OBJETIVO: Este estudo investigou a associação entre álcool e violência na população de jovens argentinos. MÉTODOS: Dados da Global School-based Student Health Survey (GSHS) de 2007, uma pesquisa representativa em termos nacionais com alunos do ensino médio, foram examinados utilizando-se modelos de regressão logística ajustados por idade. RESULTADOS: Dos 1328 alunos participantes entre 13 e 15 anos de idade, 51,9% declararam ter consumido álcool no último mês, com taxas mais elevadas entre meninos (p = 0,04) e alunos mais velhos (p < 0,01). Homens e mulheres que bebem demonstraram estar quase duas vezes mais propensos a relatar agressão física, quando em uma briga física, e pensamentos sobre violência autoinfligida do que aqueles que não bebem. Entre as pessoas que bebem, aquelas que reportaram saúde mental precária, haviam sido vítimas de bullying, fumavam, faziam uso de drogas ou abandonaram a escola sem permissão se mostraram duas vezes mais propensas ao envolvimento em atividades violentas do que outras pessoas que também bebem. CONCLUSÃO: Intervenções de saúde pública quanto à violência entre jovens adolescentes devem ser desenvolvidas em combinação com programas de educação sobre álcool.


OBJECTIVE: This study investigated the association between alcohol and violence among Argentine youth. METHODS: Data from the 2007 Argentina Global School-based Student Health Survey (GSHS), a nationally representative survey of middle school students, were examined using age-adjusted logistic regression models. RESULTS: Of the 1,328 participating students aged 13 to 15 years old, 51.9% reported drinking alcohol in the previous month, with higher rates among males (p = 0.04) and older students (p < 0.01). Both male and female drinkers were nearly twice as likely as non-drinkers to report being physically attacked, being in a physical fight, and having thoughts about self-directed violence. Among drinkers, those who reported poor mental health, were victims of bullying, used tobacco or drugs, or skipped school without permission were approximately twice as likely as other drinkers to have engaged in violent activities. CONCLUSION: Public health interventions targeting violence among young adolescents should be developed in combination with alcohol education programs.


Assuntos
Adolescente , Feminino , Humanos , Masculino , Comportamento do Adolescente/psicologia , Consumo de Bebidas Alcoólicas/epidemiologia , Vigilância da População/métodos , Violência/estatística & dados numéricos , Consumo de Bebidas Alcoólicas/psicologia , Argentina/epidemiologia , Bullying , Estudos Transversais , Saúde Mental/estatística & dados numéricos , Prevalência , Distribuição por Sexo , Fatores Sexuais , Comportamento Autodestrutivo/complicações , Violência/psicologia
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