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1.
Nat Commun ; 12(1): 801, 2021 02 05.
Artigo em Inglês | MEDLINE | ID: mdl-33547324

RESUMO

Most trials do not release interim summaries on efficacy and toxicity of the experimental treatments being tested, with this information only released to the public after the trial has ended. While early release of clinical trial data to physicians and patients can inform enrollment decision making, it may also affect key operating characteristics of the trial, statistical validity and trial duration. We investigate the public release of early efficacy and toxicity results, during ongoing clinical studies, to better inform patients about their enrollment options. We use simulation models of phase II glioblastoma (GBM) clinical trials in which early efficacy and toxicity estimates are periodically released accordingly to a pre-specified protocol. Patients can use the reported interim efficacy and toxicity information, with the support of physicians, to decide which trial to enroll in. We describe potential effects on various operating characteristics, including the study duration, selection bias and power.


Assuntos
Antineoplásicos/uso terapêutico , Neoplasias Encefálicas/psicologia , Drogas em Investigação/uso terapêutico , Glioblastoma/psicologia , Disseminação de Informação/métodos , Modelagem Computacional Específica para o Paciente , Neoplasias Encefálicas/tratamento farmacológico , Neoplasias Encefálicas/mortalidade , Neoplasias Encefálicas/patologia , Ensaios Clínicos como Assunto , Tomada de Decisões , Glioblastoma/tratamento farmacológico , Glioblastoma/mortalidade , Glioblastoma/patologia , Humanos , Disseminação de Informação/ética , Segurança do Paciente , Seleção de Pacientes/ética , Análise de Sobrevida , Fatores de Tempo , Resultado do Tratamento
2.
Drug Res (Stuttg) ; 71(4): 173-179, 2021 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-33434935

RESUMO

Coronavirus disease (COVID-19) emerged from Wuhan, has now become pandemic and the mortality rate is growing exponentially. Clinical complication and fatality rate is much higher for patients having co-morbid issues. Compromised immune response and hyper inflammation is hall mark of pathogenesis and major cause of mortality. Cytokine release syndrome (CRS) or cytokine storm is a term used to affiliate the situation of hyper inflammation and therefore use of anti-cytokine and anti-inflammatory drugs is used to take care of this situation. Looking into the clinical benefit of these anti-inflammatory drugs, many of them enter into clinical trials. However, understanding the immunopathology of COVID-19 is important otherwise, indiscriminate use of these drugs could be fetal as there exists a very fine line of difference between viral clearing cytokines and inflammatory cytokines. If any drug suppresses the viral clearing cytokines, it will worsen the situation and hence, the use of these drugs must be based on the clinical condition, viral load, co-existing disease condition and severity of the infection.


Assuntos
/tratamento farmacológico , Síndrome da Liberação de Citocina/tratamento farmacológico , Citocinas/metabolismo , Drogas em Investigação/uso terapêutico , Ativação de Macrófagos/efeitos dos fármacos , Anti-Inflamatórios/uso terapêutico , Síndrome da Liberação de Citocina/etiologia , Síndrome da Liberação de Citocina/metabolismo , Humanos , Inflamação/tratamento farmacológico , Inflamação/metabolismo
3.
Expert Opin Investig Drugs ; 30(2): 167-176, 2021 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-33393390

RESUMO

INTRODUCTION: Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder caused by mutations in the dystrophin (DMD) gene. Most patients die from respiratory failure or cardiomyopathy. There are significant unmet needs for treatments for DMD as the standard of care is principally limited to symptom relief through treatments including steroids. AREAS COVERED: This review summarizes safety and efficacy in promising areas of DMD therapeutics - small molecules, stop codon readthrough, gene replacement, and exon skipping - under clinical examination from 2015-2020 as demonstrated in the NIH Clinical Trials and PubMed search engines. EXPERT OPINION: Currently, steroids persist as the most accessible medicine for DMD. Stop-codon readthrough, gene replacement, and exon-skipping therapies all aim to restore dystrophin expression. Of these strategies, gene replacement therapy has recently gained momentum while exon-skipping retains great traction. The  FDA approval of three exon-skipping antisense oligonucleotides illustrate this regulatory momentum, though the effectiveness and sequence design of eteplirsen remain controversial. Cell-penetrating peptides promise to more efficaciously treat DMD-related cardiomyopathy.The recent success of antisense therapies, however, poses major regulatory challenges. To fully realize the benefits of exon-skipping, including cocktail oligonucleotide-mediated multiple exon-skipping and oligonucleotide drugs for very rare mutations, regulatory challenges need to be addressed in coordination with scientific advances.


Assuntos
Peptídeos Penetradores de Células/uso terapêutico , Distrofina/genética , Terapia Genética , Distrofia Muscular de Duchenne/terapia , Oligonucleotídeos/uso terapêutico , Animais , Peptídeos Penetradores de Células/efeitos adversos , Códon de Terminação , Desenvolvimento de Medicamentos , Drogas em Investigação/efeitos adversos , Drogas em Investigação/uso terapêutico , Éxons , Regulação da Expressão Gênica , Predisposição Genética para Doença , Terapia Genética/efeitos adversos , Humanos , Distrofia Muscular de Duchenne/diagnóstico , Distrofia Muscular de Duchenne/genética , Mutação , Oligonucleotídeos/efeitos adversos , Esteroides/efeitos adversos , Esteroides/uso terapêutico , Resultado do Tratamento
4.
Expert Opin Investig Drugs ; 30(2): 103-110, 2021 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-33423551

RESUMO

Introduction: Ovarian cancer (OC) represents the leading cause of death among gynecological cancers. Despite novel compound classes like vascular endothelial growth factor (VEGF) inhibitors or poly-ADP ribose polymerase (PARP) inhibitors are available, which improve significantly efficacy of platinum-based chemotherapy, OC prognosis remains poor and innovative strategies are needed. The induction of tumor specific immune response with a therapeutic intent is a very challenging approach. Oregovomab is a murine monoclonal antibody direct to the tumor-associated antigen CA125 that stimulate a host cytotoxic immune response against tumor cells expressing CA125. Areas covered: This paper reviews the preclinical and clinical published data underlying the use of oregovomab in advanced OC. A literature search was performed in PubMed for oregovomab, ovarian cancer, anti-CA125, and on ClinicalTrials.gov for currently ongoing trials. Expert opinion: Oregovomab demonstrated a significant improvement in progression-free and overall survival in advanced OC treatment when administered simultaneously with first-line chemotherapy. This promising schedule is currently investigated in a phase III trial. Since oral treatments as PARP-inhibitors have recently been approved in the OC first-line setting, the possible role of oregovomab needs still to be defined, also considering the intravenous route of administration. The easy to manage toxicity profile makes oregovomab an ideal candidate for association strategies.


Assuntos
Anticorpos Monoclonais Murinos/uso terapêutico , Antineoplásicos Imunológicos/uso terapêutico , Antígeno Ca-125/imunologia , Drogas em Investigação/uso terapêutico , Neoplasias Ovarianas/tratamento farmacológico , Animais , Anticorpos Monoclonais Murinos/efeitos adversos , Antineoplásicos Imunológicos/efeitos adversos , Drogas em Investigação/efeitos adversos , Feminino , Humanos , Neoplasias Ovarianas/diagnóstico , Neoplasias Ovarianas/imunologia , Neoplasias Ovarianas/mortalidade , Intervalo Livre de Progressão , Microambiente Tumoral
5.
Intern Emerg Med ; 16(2): 281-308, 2021 03.
Artigo em Inglês | MEDLINE | ID: mdl-33398609

RESUMO

Since its outbreak in China in December 2019 a novel Coronavirus, named SARS-CoV-2, has spread worldwide causing many cases of severe pneumonia, referred to as COVID-19 disease, leading the World Health Organization to declare a pandemic emergency in March 2020. Up to now, no specific therapy against COVID-19 disease exists. This paper aims to review COVID-19 treatment options currently under investigation. We divided the studied drugs into three categories (antiviral, immunomodulatory and other drugs). For each molecule, we discussed the putative mechanisms by which the drug may act against SARS-CoV-2 or may affect COVID-19 pathogenesis and the main clinical studies performed so far. The published clinical studies suffer from methodological limitations due to the emergency setting in which they have been conducted. Nevertheless, it seems that the timing of administration of the diverse categories of drugs is crucial in determining clinical efficacy. Antiviral drugs, in particular Remdesivir, should be administered soon after symptoms onset, in the viraemic phase of the disease; whereas, immunomodulatory agents, such as tocilizumab, anakinra and steroids, may have better results if administered in pneumonia/hyperinflammatory phases. Low-molecular-weight heparin may also have a role when facing COVID-19-related coagulopathy. Up to now, treatment choices have been inferred from the experience with other coronaviruses or viral infection outbreaks. Hopefully, in the near future, new treatment strategies will be available thanks to increased knowledge on SARS-CoV2 virus and COVID-19 pathogenesis. In the meanwhile, further well-designed clinical trials are urgently needed to establish a standard of care in COVID-19 disease.


Assuntos
Antivirais/uso terapêutico , Heparina de Baixo Peso Molecular/uso terapêutico , Fatores Imunológicos/uso terapêutico , Pneumonia Viral/tratamento farmacológico , Esteroides/uso terapêutico , /epidemiologia , Drogas em Investigação/uso terapêutico , Humanos , Pandemias , Pneumonia Viral/epidemiologia , Pneumonia Viral/virologia
6.
BMJ Case Rep ; 13(12)2020 Dec 17.
Artigo em Inglês | MEDLINE | ID: mdl-33334744

RESUMO

Myxopapillary ependymoma (MPE) is a rare glial tumour mainly located in the areas of the conus medullaris, cauda equina and filum terminale of the spinal cord. Ectopic MPE tends to behave more aggressively and distant metastases are often seen. Unfortunately, no standard treatment options are established as only small series of treated patients and a few reported cases are available in the literature. We report the case of a 25-year-old woman who was initially diagnosed with a metastatic MPE, with multiple bilateral lung metastases. She was treated with an investigational monoclonal antibody antiprogrammed cell death protein 1, called tislelizumab (BGB-A317), following surgical resection of the perisacral primary mass. The response was long-lasting and side effects nil. Immunotherapy is a treatment modality to be considered in patients with rare tumours.


Assuntos
Anticorpos Monoclonais Humanizados/uso terapêutico , Drogas em Investigação/uso terapêutico , Ependimoma/terapia , Neoplasias Pulmonares/terapia , Neoplasias da Medula Espinal/terapia , Biópsia com Agulha de Grande Calibre , Quimioterapia Adjuvante/métodos , Ependimoma/complicações , Ependimoma/diagnóstico , Ependimoma/secundário , Feminino , Humanos , Dor Lombar/etiologia , Dor Lombar/terapia , Neoplasias Pulmonares/complicações , Neoplasias Pulmonares/diagnóstico , Neoplasias Pulmonares/secundário , Imagem por Ressonância Magnética , Invasividade Neoplásica , Sacro/diagnóstico por imagem , Sacro/patologia , Sacro/cirurgia , Medula Espinal/patologia , Medula Espinal/cirurgia , Neoplasias da Medula Espinal/complicações , Neoplasias da Medula Espinal/diagnóstico , Neoplasias da Medula Espinal/patologia , Tomografia Computadorizada por Raios X , Resultado do Tratamento , Adulto Jovem
8.
BMJ ; 370: m3176, 2020 09 21.
Artigo em Inglês | MEDLINE | ID: mdl-32958461

RESUMO

Despite considerable advances in treatment approaches in the past two decades, multiple myeloma remains an incurable disease. Treatments for myeloma continue to evolve with many emerging immunotherapies. The first immunotherapy used to treat hematologic cancers, including multiple myeloma, was an allogeneic stem cell transplant. In the mid-2000s, immunomodulatory drugs thalidomide, lenalidomide, and subsequently pomalidomide were proven to be effective in multiple myeloma and substantially improved survival. The next wave of immunotherapies for multiple myeloma included the monoclonal antibodies daratumumab and elotuzumab, which were approved by the Food and Drug Administration in 2015. Subsequently, a variety of immunotherapies have been developed for multiple myeloma, including chimeric antigen receptor T cells, bispecific antibodies, antibody drug conjugates, and checkpoint inhibitors. Many of these emerging treatments target the B cell maturation antigen, which is expressed on plasma cells, although several other novel receptors are also being studied. This review summarizes the evidence of these various immunotherapies, their mechanism of action, and data from clinical trials regarding the treatments' safety and efficacy.


Assuntos
Imunoterapia , Mieloma Múltiplo/terapia , Drogas em Investigação/uso terapêutico , Humanos , Fatores Imunológicos/uso terapêutico , Transplante de Células-Tronco
11.
Ann Hematol ; 99(7): 1429-1440, 2020 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-32514626

RESUMO

With the advent of new targeted drugs in hematology and oncology patient prognosis is improved. Combination with antifungal prophylaxis challenges clinicians due to pharmacological profiles prone to drug-drug interactions (DDI). Midostaurin is a novel agent for FLT3-TKD/-ITDmut-acute myeloid leukemia (AML) and metabolized via cytochrome P450 3A4 (CYP3A4). Posaconazole is a standard of care antifungal agent used for prophylaxis during induction treatment of AML and a strong CYP3A4 inhibitor. Concomitant administration of both drugs leads to elevated midostaurin exposure. Both drugs improve overall survival at low numbers needed to treat. The impact of CYP3A4-related DDI remains to be determined. Severe adverse events have been observed; however, it remains unclear if they can be directly linked to DDI. The lack of prospective clinical studies assessing incidence of invasive fungal infections and clinical impact of DDI contributes to neglecting live-saving antifungal prophylaxis. Management strategies to combine both drugs have been proposed, but evidence on which approach to use is scarce. In this review, we discuss several approaches in the specific clinical setting of concomitant administration of midostaurin and posaconazole and give examples from everyday clinical practice. Therapeutic drug monitoring will become increasingly important to individualize and personalize antineoplastic concomitant and antifungal treatment in the context of DDI. Pharmaceutical companies addressing the issue in clinical trials may take a pioneer role in this field. Other recently developed and approved drugs for the treatment of AML likely inhere potential of DDI marking a foreseeable issue in future treatment of this life-threatening disease.


Assuntos
Antifúngicos/uso terapêutico , Quimioprevenção/tendências , Infecções Fúngicas Invasivas/prevenção & controle , Leucemia Mieloide Aguda/tratamento farmacológico , Estaurosporina/análogos & derivados , Triazóis/uso terapêutico , Antifúngicos/classificação , Quimioprevenção/métodos , Interações Medicamentosas , Drogas em Investigação/uso terapêutico , Humanos , Leucemia Mieloide Aguda/diagnóstico , Leucemia Mieloide Aguda/microbiologia , Leucemia Mieloide Aguda/mortalidade , Prognóstico , Estaurosporina/uso terapêutico
12.
In Vivo ; 34(3 Suppl): 1597-1602, 2020 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-32503817

RESUMO

Severe acute respiratory syndrome coronavirus 2 (SARS-Cov-2), initially termed 2019-new CoV (2019-nCoV), is a novel coronavirus responsible for the severe respiratory illness currently ongoing worldwide from the beginning of December 2019. This beta gene virus, very close to bat coronaviruses (bat-CoV-RaTG13) and bat-SL-CoVZC45, causes a severe disease, similar to those caused by Middle East respiratory syndrome (MERS)-CoV and SARS-CoV viruses, featured by low to moderate mortality rate. Unfortunately, the antiviral drugs commonly used in clinical practice to treat viral infections, are not applicable to SARS-Cov-2 and no vaccine is available. Thus, it is extremely necessary to identify new drugs suitable for the treatment of the 2019-nCoV outbreak. Different preclinical studies conducted on other coronaviruses suggested that promising clinical outcomes for 2019-nCoV should be obtained by using alpha-interferon, chloroquine phosphate, arabinol, remdesivir, lopinavir/ritonavir, and anti-inflammatory drugs. Moreover, clinical trials with these suitable drugs should be performed on patients affected by SARS-Cov-2 to prove their efficacy and safety. Finally, a very promising therapeutic drug, tocilizumab, is discussed; it is currently used to treat patients presenting COVID-19 pneumonia. Herein, we recapitulate these experimental studies to highlight the use of antiviral drugs for the treatment of SARS-Cov-2 disease.


Assuntos
Antivirais/uso terapêutico , Infecções por Coronavirus/tratamento farmacológico , Pandemias , Pneumonia Viral/tratamento farmacológico , Monofosfato de Adenosina/análogos & derivados , Monofosfato de Adenosina/uso terapêutico , Alanina/análogos & derivados , Alanina/uso terapêutico , Animais , Anticorpos Monoclonais Humanizados/uso terapêutico , Antivirais/administração & dosagem , Antivirais/farmacologia , Betacoronavirus/efeitos dos fármacos , Cloroquina/análogos & derivados , Cloroquina/uso terapêutico , Ensaios Clínicos como Assunto , Infecções por Coronavirus/complicações , Infecções por Coronavirus/epidemiologia , Infecções por Coronavirus/veterinária , Combinação de Medicamentos , Avaliação Pré-Clínica de Medicamentos , Sinergismo Farmacológico , Quimioterapia Combinada , Drogas em Investigação/uso terapêutico , Humanos , Indóis/uso terapêutico , Lopinavir/uso terapêutico , Estudos Multicêntricos como Assunto , Neuraminidase/antagonistas & inibidores , Pneumonia Viral/complicações , Pneumonia Viral/epidemiologia , Primatas , Ribavirina/uso terapêutico , Ritonavir/uso terapêutico , Resultado do Tratamento
13.
Farm Hosp ; 44(7): 66-70, 2020 06 13.
Artigo em Inglês | MEDLINE | ID: mdl-32533675

RESUMO

The health crisis resulting from the rapid spread of SARS-CoV-2 worlwide, added to the low evidence of currently used treatments has led to the development of a large number of clinical trials (CT) and observational studies. Likewise,  important measures have been adopted in healthcare and research centers  aimed at halting the pandemic as soon as possible. The objective of this study is  to gather the main aspects of the clinical research studies undertaken by the  Departments of Hospital Pharmacy (DHP) of Spain during the COVID-19 crisis. The decision of the Spanish Society of Hospital Pharmacy (SEFH) to sponsor CTs made it possible that 13% of DHP had been led at least one CT.  The Spanish Agency for Medicines and Medical Devices (AEMPS), in coordination  with Institutional Review Boards, has adopted a fast-track review procedure to  accelerate authorizations for CTs related to the treatment or prevention of  COVID-19. There have also been numerous public and private calls for financing  research projects aimed at contributing to the fight against this virus. Despite  the pandemic, actions have been taken to continue ongoing CTs and studies  while the safety and well-being of patients are guaranteed. More specifically, the AEMPS and the European Medicines Agency (EMA) have issued guidelines that  incorporate changes to CT protocols that will have to be applied until the  pandemic is over. In this health emergency, the scientific community has found  itself in a race against time to generate evidence. It is at this moment that  hospital pharmacists emerge as key players in clinical research and are  contributing to a rational, effective and safe healthcare decision-making.


Assuntos
Betacoronavirus , Ensaios Clínicos como Assunto , Infecções por Coronavirus/tratamento farmacológico , Controle de Infecções/organização & administração , Estudos Multicêntricos como Assunto , Estudos Observacionais como Assunto , Pandemias , Serviço de Farmácia Hospitalar/organização & administração , Pneumonia Viral/tratamento farmacológico , Antivirais/uso terapêutico , Ensaios Clínicos como Assunto/economia , Ensaios Clínicos como Assunto/estatística & dados numéricos , Infecções por Coronavirus/epidemiologia , Infecções por Coronavirus/prevenção & controle , Tomada de Decisões , Drogas em Investigação/uso terapêutico , Previsões , Humanos , Estudos Multicêntricos como Assunto/economia , Estudos Multicêntricos como Assunto/estatística & dados numéricos , Estudos Observacionais como Assunto/economia , Estudos Observacionais como Assunto/estatística & dados numéricos , Pandemias/prevenção & controle , Segurança do Paciente , Pneumonia Viral/epidemiologia , Pneumonia Viral/prevenção & controle , Projetos de Pesquisa , Apoio à Pesquisa como Assunto , Papel (figurativo) , Espanha
14.
Acta Med Port ; 33(7-8): 500-504, 2020 Jul 01.
Artigo em Inglês | MEDLINE | ID: mdl-32425152

RESUMO

The novel severe acute respiratory syndrome coronavirus 2 is the cause of Coronavirus Disease 2019, a new illness with no effective treatment or vaccine that has reached pandemic proportions. In this document, we analyze how health authorities and agencies around the world position themselves regarding the off-label use of repurposed drugs or new investigational drugs to treat Coronavirus Disease 2019. We review the most promising candidate medicines, including available evidence, clinical recommendations and current options for access. Our concluding remarks stress the importance of administering off-label and investigational drugs in the setting of clinical trials, or at least in standardized scenarios, to generate as much scientific knowledge as achievable while engaging in the best efforts to treat patients and save lives.


Assuntos
Antivirais/uso terapêutico , Betacoronavirus , Infecções por Coronavirus/tratamento farmacológico , Drogas em Investigação/uso terapêutico , Uso Off-Label , Pneumonia Viral/tratamento farmacológico , Monofosfato de Adenosina/análogos & derivados , Monofosfato de Adenosina/uso terapêutico , Alanina/análogos & derivados , Alanina/uso terapêutico , Anticorpos Monoclonais Humanizados/uso terapêutico , Cloroquina/uso terapêutico , Infecções por Coronavirus/epidemiologia , Combinação de Medicamentos , Saúde Global , Humanos , Hidroxicloroquina/uso terapêutico , Lopinavir/uso terapêutico , Pandemias , Pneumonia Viral/epidemiologia , Ritonavir/uso terapêutico
15.
Encephale ; 46(3S): S114-S115, 2020 Jun.
Artigo em Francês | MEDLINE | ID: mdl-32362504

RESUMO

The analysis of real-life data from hospital information systems could make possible to decide on the efficacy and safety of Covid-19 treatments by avoiding the pitfalls of preliminary studies and randomized clinical trials. The different drugs tested in current clinical trials are already widely prescribed to patients by doctors in hospitals, and can therefore be immediately analysed according to validated methodological standards.


Assuntos
Betacoronavirus , Infecções por Coronavirus/epidemiologia , Sistemas de Informação Hospitalar/estatística & dados numéricos , Registros Hospitalares/estatística & dados numéricos , Pandemias , Pneumonia Viral/epidemiologia , Projetos de Pesquisa , Antivirais/uso terapêutico , Infecções por Coronavirus/tratamento farmacológico , Reposicionamento de Medicamentos , Drogas em Investigação/uso terapêutico , Medicina Baseada em Evidências , França/epidemiologia , Humanos , Hidroxicloroquina/uso terapêutico , Pneumonia Viral/tratamento farmacológico , Ensaios Clínicos Controlados Aleatórios como Assunto/estatística & dados numéricos , Estudos Retrospectivos , Software
18.
J Clin Psychiatry ; 81(3)2020 04 14.
Artigo em Inglês | MEDLINE | ID: mdl-32297721

RESUMO

The currently available antipsychotics mostly treat the positive symptoms of schizophrenia and have at least one adverse effect as a potential liability. Encouraging data suggest potential efficacy for a variety of new agents for the treatment of total symptoms and/or specific symptom domains of schizophrenia. Mechanisms of action that are under investigation include dopamine D3 antagonism/serotonin 5-HT1A partial agonism; combined dopamine, serotonin, and glutamate modulation; phosphodiesterase 10A inhibition; trace amine-associated receptor-1 (TAAR1) agonism plus 5-HT1A agonism; 5-HT2A inverse agonism; sigma-2/5-HT2A antagonism; D-amino acid oxidase (DAAO) inhibition; glycine transporter-1 inhibition; vesicular monoamine transporter-2 antagonism; mu opioid antagonism added to olanzapine; and novel long-acting injectable antipsychotic formulations. It is hoped that ongoing and recently completed trials for agents with known and/or novel mechanisms of action will lead to approved treatments that effectively target the various symptom domains of schizophrenia, minimize the risk for a broad range of clinically relevant adverse effects, and improve functional outcomes for patients. Some novel treatments have already received approval for use in patients with schizophrenia. This brief report discusses recently approved novel agents and potential new treatment options for schizophrenia that are being investigated.


Assuntos
Antipsicóticos/uso terapêutico , Esquizofrenia/tratamento farmacológico , Drogas em Investigação/uso terapêutico , Humanos
20.
Expert Opin Pharmacother ; 21(7): 841-851, 2020 May.
Artigo em Inglês | MEDLINE | ID: mdl-32133879

RESUMO

INTRODUCTION: Duchenne muscular dystrophy (DMD) is the result of X-chromosome-linked mutations to the dystrophin protein gene that prevent the normal development and repair of muscles leading to muscle deterioration. The condition affects nearly 1 in 3,500 males worldwide. Current therapeutics have not been sufficient in providing a cure or resulting in a significant extension in life expectancy, but many therapeutic options are currently under investigation. AREAS COVERED: This article provides an overview of the current and emerging therapies for DMD giving particular focus to synthetic therapeutic options. The authors further provide their expert opinion. EXPERT OPINION: Many discrepancies in primary outcomes of trials have led to questions of efficacy for medications, as well as difficulty in securing FDA approval. A standardization of primary outcome strategies, as well as better access to investigational medications, may alleviate some of the controversy and pressures that exist on medication approvals. Many trials have identified cohorts who responded more favorably to medications, despite a lack of significance in the overall intent-to-treat populations. This indicates that more medication screening and personalized treatment with patient-specific targeting might deliver more clinically significant results.


Assuntos
Antibacterianos/uso terapêutico , Anti-Inflamatórios/uso terapêutico , Drogas em Investigação/uso terapêutico , Distrofina/genética , Distrofia Muscular de Duchenne/tratamento farmacológico , Animais , Ensaios Clínicos como Assunto , Terapia Genética , Humanos , Masculino , Distrofia Muscular de Duchenne/genética , Mutação , Transplante de Células-Tronco
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