Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies.

abstract：IMPORTANCE OF THE FIELD:Irritable bowel syndrome (IBS) represents one of the most common gastrointestinal disorders, with the diarrhea-predominant form (D-IBS) representing an area of high unmet medical need. One difficulty in identifying suitable treatments for IBS is that although there are animal models for the comp...

journal_title：Expert opinion on investigational drugs

authors： Mangel AW,Williams VS

更新日期：2010-10-01 00:00:00

abstract：:Gossypol has received significant attention as a result of its potential therapeutic application as a male antifertility agent. Furthermore, recent research examining the biological activity of gossypol has revealed a number of other promising lines of enquiry. These have focused on the antitumour, antiviral and antio...

journal_title：Expert opinion on investigational drugs

authors： Dodou K,Anderson RJ,Small DA,Groundwater PW

更新日期：2005-11-01 00:00:00

abstract：INTRODUCTION:The N-methyl-d-aspartate receptor-ionophore complex plays a key role in learning and memory and has efficacy in animals and humans with affective disorders. GLYX-13 is an N-methyl-d-aspartate receptor (NMDAR) glycine-site functional partial agonist and cognitive enhancer that also shows rapid antidepressan...

journal_title：Expert opinion on investigational drugs

authors： Moskal JR,Burch R,Burgdorf JS,Kroes RA,Stanton PK,Disterhoft JF,Leander JD

更新日期：2014-02-01 00:00:00

abstract：INTRODUCTION:Therapeutic options for mycosis fungoides and Sézary syndrome include a variety of immunomodulatory, epigenetic, and cytotoxic options; however, none has been demonstrated to be efficacious for all patients, or to deliver deep and durable responses to the majority of patients. In this review, we examine th...

journal_title：Expert opinion on investigational drugs

authors： Van Der Weyden C,Bagot M,Neeson P,Darcy PK,Prince HM

更新日期：2018-08-01 00:00:00

abstract：INTRODUCTION:Melanoma is one of the most aggressive forms of cutaneous malignancies displaying a substantial mortality rate among the various forms of skin cancers. The management of patients with advanced melanoma poses a significant challenge considering that the disease is refractory to most conventional therapies. ...

journal_title：Expert opinion on investigational drugs

authors： Bhatia S,Emdad L,Das SK,Hamed H,Dent P,Sarkar D,Fisher PB

更新日期：2014-04-01 00:00:00

abstract：:Innate immune mechanisms respond rapidly to bacterial infection. A key cellular component of the innate immune response is the neutrophil, whose cytoplasmic granules contain a variety of antimicrobial proteins and peptides. Among these is the bactericidal/permeability-increasing protein (BPI), a cationic 55 kDa protei...

journal_title：Expert opinion on investigational drugs

authors： Levy O

更新日期：2002-02-01 00:00:00

abstract：INTRODUCTION:Alzheimer's disease (AD) is the most common cause of dementia in the elderly. Pharmacological treatment of AD includes Anticholinesterase Inhibitors (AChEIs) for mild-moderate AD, and memantine for severe AD. These drugs provide mainly symptomatic short-term benefits without clearly influencing the progres...

journal_title：Expert opinion on investigational drugs

authors： Galimberti D,Scarpini E

更新日期：2017-01-01 00:00:00

abstract：:Despite advances in supportive care, septic shock remains a major cause of morbidity and mortality. With the identification of the systemic inflammatory response as a major component in the pathogenesis of the septic shock syndrome, much of the recent work has focused on modulating this response. This includes antiend...

journal_title：Expert opinion on investigational drugs

authors： Sharma VK,Dellinger RP

更新日期：2003-02-01 00:00:00

abstract：:The interaction between the urokinase receptor (uPAR) and its ligand urokinase (uPA) mediates phenomena such as tissue remodelling, chemotaxis, tumour invasion, dissemination, proliferation, and angiogenesis. The broad-spectrum of biological processes that the uPA/uPAR interaction plays a role in has led researchers t...

journal_title：Expert opinion on investigational drugs

authors： Weidle UH,König B

更新日期：1998-03-01 00:00:00

abstract：INTRODUCTION:The introduction of therapy with tumor necrosis factor antagonists (aTNF) was a cornerstone of treatment modalities in patients with ankylosing spondylitis (AS). After > 10 years of using aTNF, the introduction of aTNF therapy was a major step forward in the medical management of patients with spondyloarth...

journal_title：Expert opinion on investigational drugs

authors： Braun J,Baraliakos X,Heldmann F,Kiltz U

更新日期：2014-05-01 00:00:00

abstract：BACKGROUND:Clusterin is a stress-induced cytoprotective chaperone protein, regulated by HSF1, and functions similarly to a small heat-shock protein. Clusterin is expressed in a variety of cancers and associated with broad-spectrum treatment resistance. Custirsen (OGX-011) is a 2'-methoxyethyl modified phosphorothioate ...

journal_title：Expert opinion on investigational drugs

authors： Chi KN,Zoubeidi A,Gleave ME

更新日期：2008-12-01 00:00:00

abstract：INTRODUCTION:Myocardial fibrosis is a remarkably dynamic process mediated by different molecular pathways that represent potential targets of novel therapeutic interventions. Transforming Growth Factor-beta (TGF-β), connective Tissue Growth Factor (cTGF) and Galectin-3 (Gal-3) represent the most promising targets on wh...

journal_title：Expert opinion on investigational drugs

authors： Spoladore R,Falasconi G,Fiore G,Di Maio S,Preda A,Slavich M,Margonato A,Fragasso G

更新日期：2021-01-04 00:00:00

abstract：INTRODUCTION:Progesterone receptor (PR) agonists (progestins) and antagonists are developed for female contraceptives. However, non-contraceptive applications of newer progestins and PR modulators are being given more attention. AREAS COVERED:The newer PR agonists including drospirenone, nomegestrol, trimegestone, die...

journal_title：Expert opinion on investigational drugs

authors： Su Y,Lian QQ,Ge RS

更新日期：2012-01-01 00:00:00

abstract：IMPORTANCE OF THE FIELD:Hepatitis C virus (HCV) is the main agent of chronic hepatitis, liver cirrhosis and hepatocellular carcinoma in the western world. It affects more than 170 million people worldwide. HCV treatment, based on the combination of Peg-interferon and ribavirin, is effective in about 50% of treated pati...

journal_title：Expert opinion on investigational drugs

authors： Gentile I,Carleo MA,Borgia F,Castaldo G,Borgia G

更新日期：2010-01-01 00:00:00

abstract：INTRODUCTION:The purpose of this article is to highlight novel therapies that are being used in scleroderma (SSc). Therapeutic interventions in SSc generally target at least one of three ongoing biological processes characteristic of the disease: vasculopathy, autoimmunity and tissue fibrosis. Treatment decisions in SS...

journal_title：Expert opinion on investigational drugs

authors： McMahan ZH,Wigley FM

更新日期：2014-02-01 00:00:00

abstract：INTRODUCTION:Asthma is a major disease burden worldwide. Treatment with steroids and long acting β-agonists effectively manage symptoms in many patients but do not treat the underlying cause of disease and have serious side effects when used long term and in children. Therapies targeting the underlying causes of asthma...

journal_title：Expert opinion on investigational drugs

authors： Hansbro PM,Scott GV,Essilfie AT,Kim RY,Starkey MR,Nguyen DH,Allen PD,Kaiko GE,Yang M,Horvat JC,Foster PS

更新日期：2013-01-01 00:00:00

abstract：IMPORTANCE OF THE FIELD:Molecules isolated from animals, insects, plants or microorganisms can provide prototypes for design of biopharmaceutical products. Some venom toxins and their derivatives are used in medicine, while others provide templates for development of new drugs. AREAS COVERED IN THIS REVIEW:The mild to...

journal_title：Expert opinion on investigational drugs

authors： Kerkis I,Silva Fde S,Pereira A,Kerkis A,Rádis-Baptista G

更新日期：2010-12-01 00:00:00

abstract：INTRODUCTION:Although significant advances have been made in the treatment of advanced renal cell carcinoma (RCC), patients still develop resistance to standard therapies and require the administration of subsequent lines of treatment. New therapeutic approaches are thus imperative to improve the prognosis for patients...

journal_title：Expert opinion on investigational drugs

authors： Bilen MA,Carlisle JW,Sonpavde G

更新日期：2018-02-01 00:00:00

abstract：:Neural cell transplantation is an emerging therapy that may provide an effective treatment for neurodegenerative disorders. The most extensive work with neural transplants has been carried out for Parkinson's and Huntington's diseases. However, intensive efforts are also being made for the treatment of other neurologi...

journal_title：Expert opinion on investigational drugs

authors： Dinsmore JH

更新日期：1998-04-01 00:00:00

abstract：:Clinical trials of several platelet glycoprotein (GP) IIb/IIIa receptor inhibitors have demonstrated an unequivocal benefit of this potent antithrombotic therapy in high-risk patients with acute coronary syndromes (ACS) as well as in those undergoing percutaneous coronary intervention. In all of these major trials, ho...

journal_title：Expert opinion on investigational drugs

authors： Salam AM

更新日期：2003-09-01 00:00:00

abstract：:Gene transfer technology is under exploration to find therapies for the treatment of Parkinson's disease (PD) and other neurodegenerative disorders. The technology of genetic transfer can also be used as a neurobiological tool to understand the role of various genes in animal models of neurodegeneration. We describe t...

journal_title：Expert opinion on investigational drugs

authors： Frim DM,Kang UJ

更新日期：1999-10-01 00:00:00

abstract：:Two classes of luteinising hormone-releasing hormone (LHRH) analogues have been developed so far to be used for oncological therapies: LHRH agonists and antagonists. LHRH agonists are widely and successfully used for the management of steroid-dependent malignancies. Chronic administrations of these compounds result in...

journal_title：Expert opinion on investigational drugs

authors： Limonta P,Montagnani Marelli M,Moretti RM

更新日期：2001-04-01 00:00:00

abstract：INTRODUCTION:Angiogenesis inhibition represents a rational therapeutic strategy in the management of solid tumors. Brivanib is a dual tyrosine kinase inhibitor with selectivity against VEFGR-2 and FGFR. AREAS COVERED:This review provides an updated summary of preclinical and clinical experience with brivanib in cancer...

journal_title：Expert opinion on investigational drugs

authors： Diaz-Padilla I,Siu LL

更新日期：2011-04-01 00:00:00

abstract：:Aripiprazole has made a significant contribution to the treatment of schizophrenia and related disorders with an improved safety and tolerability profile, which has been attributed to its unique pharmacological profile. It has been claimed that partial agonism of the dopamine D(2) and 5-HT(1A) receptors and antagonism...

journal_title：Expert opinion on investigational drugs

authors： Wood M,Reavill C

更新日期：2007-06-01 00:00:00

abstract：:The annual meeting of the American Diabetes Association was held in Boston, MA. There were over 6500 participants, one-third from overseas, and 1103 papers were presented. Abstracts from the meeting have been published, from which abstract numbers are given below, e.g., (A141). Considerable progress has been made in u...

journal_title：Expert opinion on investigational drugs

authors： Colca J

更新日期：1997-08-01 00:00:00

abstract：BACKGROUND:In the past years the bone seeking radiopharmaceutical samarium lexidronam ((153)Sm-EDTMP) has been increasingly used alone or in conjunction with chemotherapy and/or bisphosphonates for the treatment of painful bone metastasis. OBJECTIVE:Its use has been explored in different solid tumours. In this report ...

journal_title：Expert opinion on investigational drugs

authors： Abruzzese E,Iuliano F,Trawinska MM,Di Maio M

更新日期：2008-09-01 00:00:00

abstract：INTRODUCTION:Rheumatoid arthritis (RA) is a chronic inflammatory auto-immune disease that can lead to permanent disability and deformity. Despite current treatment modalities, many patients are still unable to reach remission. Interleukin-1 receptor-associated kinase 4 (IRAK-4) inhibitors are novel agents designed to s...

journal_title：Expert opinion on investigational drugs

authors： Wiese MD,Manning-Bennett AT,Abuhelwa AY

更新日期：2020-05-01 00:00:00

abstract：INTRODUCTION:Asthma is a common respiratory disease characterized by airway inflammation, bronchoconstriction and airway hyperresponsiveness and symptoms such as coughing, wheezing, shortness of breath and chest tightness. Allergic rhinitis is a common comorbidity in asthma and glucocorticoids are the key stone in the ...

journal_title：Expert opinion on investigational drugs

authors： Meteran H,Backer V

更新日期：2016-08-01 00:00:00

abstract：:COPD is a chronic disease of the lung that is characterised by decreased air flow and associated abnormal inflammatory responses of the lungs. A total of 80% of COPD incidences are observed in patients with history of smoking tobacco. The chronic condition of COPD is characterised by airway remodelling, which leads to...

journal_title：Expert opinion on investigational drugs

authors： Srivastava PK,Dastidar SG,Ray A

更新日期：2007-07-01 00:00:00

abstract：INTRODUCTION:Patients with myelofibrosis (MF) have no effective treatment option after the failure of approved JAK inhibitor (JAKi) therapy. Non-JAK inhibitors (non-JAKi) that target non-canonical molecular pathways are undergoing clinical evaluations to optimize efficacy and/or to reduce hematological toxicity of JAKi...

journal_title：Expert opinion on investigational drugs

authors： Bankar A,Gupta V

更新日期：2020-05-01 00:00:00