Clostridium Difficile- Associated Diarrhea Therapeutics Pipeline Analysis 2017
The prevalence of clostridium difficile associated diarrhea has been increasing all across the world on account of unchecked use of antibiotics and laxatives by people, which creates a suitable medium for the growth of the bacteria. Clostridium Difficile is a commonly noticed disease in people above 65 years of age, as they tend to consume antibiotics and laxatives for easy and effective bowel movement. The therapeutic pipeline of clostridium difficile associated diarrhea is growing with the involvement of various pharmaceutical companies that are working towards the development of drugs or therapeutic agentsto be used for the treatment of clostridium difficile associated diarrhea. According to a paper published by NCBI, in 2012, clostridium difficile associated diarrhea is responsible for causing diarrhea to 3 million people in the U.S. Clostridium difficile associated diarrhea (CDAD) is also caused by nosocomial infection, acquired from hospitals due to presence of different viruses and pathogens in the hospitals. It is projected that the number of cases related to clostridium difficile associated diarrhea would increase in the future, with increase in unrestrained consumption of antibiotics.
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Clostridium difficile is a rod-shaped gram positive bacterium, which is responsible for causing diarrhea. It is the most common type of infection, and is usually found more in developing countries such as India, Iran, Pakistan, Afghanistan, Iraq, Nepal and China. Some of the symptoms of clostridium difficile associated diarrhea include severe colitis, fever, abdominal pain, dehydration, rapid heart rate, nausea, blood in the stool and loss of appetite. Clostridium difficile has been reported as more common in women and older patients.
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Some of the companies having a pipeline of clostridium difficile associated diarrhea (CDAD) therapeutics include Astella pharma Inc.,Merck Sharp & Dohme Corp,Actelion Inc., Da volterra Inc.,Eliy-lilly& Company, Gilead Sciences, Inc., Galapagos NV, Astellas Pharma, Abbvie, Inc., Bexalta US, Inc., Optimer pharmaceutical LLC.
Fragile-x Syndrome Therapeutics Pipeline Analysis 2017 - Designation, Collaboration, and Other Developments
Due to lack of awareness among people regarding genetic disorders which leads to mutations in one’s DNA, the prevalence of fragile-x syndrome is increasing, across the globe. Smoking, alcohol consumption during pregnancy and hereditary disorders are few of the primary reasons to for the increase in prevalence of fragile-x syndrome. The therapeutic pipeline of fragile-x syndrome is expected to grow more rapidly in the future on account of active involvement of pharmaceuticals & biotechnology companies carrying out research programs to develop effective therapeutic agents or drugs for eradicating fragile-x syndrome. According to a study conducted by Centers for Disease Control and Prevention (CDC) in 2012, around one female in every 151 females & one male in every 468 males in the U.S., was affected by fragile-x syndrome. Moreover, according to the study, occurrence of fragile-x syndrome is higher in females as compared to males.
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Fragile-x syndrome is a genetic disorder that affects the development and learning capabilities of an individual. It is also called as Marker-x syndrome and Martin-Bell syndrome. Fragile-x syndrome causes trinucleotide repeat in Fragile-x mental retardation 1(FMR-1) gene present on X chromosome. This gene is responsible for making a protein called Fragile-x mental retardation protein (FMRP), which is required for normal neural development. In case of an individual suffering from fragile-x syndrome, production of FMRP is constrained due to the repetition of CGG (trinucleotide) in FMR1 gene. The symptoms associated with fragile-x syndrome are mental retardation, learning disability, impulsiveness, stuttering, development delays, seizures, depression, hyperactivity, etc.
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Some of the companies having a pipeline of Fragile-x syndrome therapeutics include Ovid therapeutics, Marinus pharmaceuticals, Neuren pharmaceuticals, Bellus health Inc., AMO pharmaceuticals, Eli-lly& co., Anavex Life Sciences Corp., Kareus Therapeutics, and Zynerba Pharmaceuticals, Inc.
Phosphatidylinositol 3-Kinase Inhibitors Pipeline Analysis 2017
The PI3K inhibitors pipeline analysis report covers approximately 37 active drug candidates in different stages of development.
Phosphatidylinositol 3-kinases (PI3K) are lipid kinases that are involved in cell regulation, including cell survival, proliferation and differentiation. They act as intermediate molecules in PI3K/AKT/mTOR signaling pathway by sending chemical messengers from cell surface to cytoplasm. These signals activate multiple effector kinase pathways, resulting in survival and growth of normal cells. Phosphatidylinositol 3-kinases is categorized on the basis of classes as class I, II and III, on the basis of structure of the PI3K, based on the specificities of the substrate as well as on the basis of lipid end-product’s nature. Bases on structure, Phosphatidylinositol 3-kinases has four different forms PI3K alpha, PI3K beta, PI3K gamma and PI3K delta.
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In March 2017, Bayer AG presented clinical results of Phosphoinositide 3-kinase (PI3K)-targeting drug candidate Copanlisib in Phase II CHRONOS-1 trial, evaluating patients with relapsed or refractory indolent non-Hodgkin's lymphoma (iNHL), trial with NCT ID 01660451. The results across all patient groups showed an objective response rate (ORR) of 59.2%, with a 12% complete response (CR) rate and a median duration of response (DOR) of more than 98 weeks, or 687 days (range 0-687).
The research found that different companies engaged in manufacturing of PI3K inhibitors are collaborating. One of the collaborations occurred in November 2016 between Verastem, Inc. and Infinity Pharmaceuticals, Inc.
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The major players for PI3K inhibitors pipeline include, but are not limited to, Novartis AG, Genentech, Inc., SignalRX Pharmaceuticals Inc. and others.
Nasopharyngeal Cancer Therapeutics Pipeline - Collaboration and Other Developments
Nasopharyngeal cancer therapeutic pipeline analysis is likely to grow mainly due to the rising prevalence of nasopharyngeal cancer, globally. Some of the main factors driving the pipeline analysis for nasopharyngeal cancer includes increasing geriatric population, rising tobacco and alcohol consumption and increasing prevalence of nasopharyngeal cancer, untapped market as being a rare form of head and neck cancer and increasing health awareness. According to American Cancer Society, the nasopharyngeal cancer is more common in some parts of North Africa and Asia, particularly in Southern China. It is also more common in countries such as Canada and Alaska and among some immigrant groups in the U.S., such as recent Chinese immigrants. Nasopharyngeal cancer is found to be more prevalent in males as compared to females.
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Nasopharyngeal cancer is the rarest form of head and neck cancer. Nasopharyngeal cancer is the tumor that occurs in nasopharynx which is the upper part of the throat behind the nose and near the base of skull. Two different types of tumors that can develop in nasopharynx include benign tumors and malignant tumors. Some of the risks factors for nasopharyngeal cancer include salt-cured fish and meat rich diet, epstein-barr viral infection and increasing tobacco and alcohol consumption. The disease occurs usually more in people who are under 55years of age and have a family history of nasopharyngeal cancer or have the chances to develop Epstein-barr viral infection. The symptoms of nasopharyngeal cancer include blurred vision, ear infection, headache, nose bleeding, lump in neck and numbness. Nasopharyngeal cancer is diagnosed by consulting an otolaryngologist for the physical examination. Further, chest X-ray, CT scan, MRI, ultrasound, blood tests, Epstein-barr viral infection testing and biopsy are done for the confirmation of the disease. The treatment for nasopharyngeal cancer includes, chemotherapy, radiation therapy, surgery and biological drug treatment.
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Some of the companies having a pipeline of nasopharyngeal cancer therapeutics include Atara Biotherapeutics, Inc., AVEO Pharmaceuticals, Inc., Novartis AG, Ono Pharmaceutical Co., Ltd., F. Hoffmann-La Roche Ltd., GlaxoSmithKline Plc, arGEN-X BVBA, Merck & Co., Inc., Tessa Therapeutics Pte Ltd.
Spinal Muscular Atrophy Therapeutics Pipeline Analysis 2017
The study analyzed that the therapeutics pipeline comprises approximately 16 drug candidates in different stages of development. Spinal muscular atrophy is defined as the inherited genetic disease that is characterized by a failure of nerve cells called motor neurons. Motor neurons are responsible to accept the nerve impulses transmitted from the brain to the spinal cord and transmit the impulses to the muscle with the help of peripheral nerves. The loss of motor neurons leads to muscle weakness in muscles that are closest to the trunk of the body such as back, hips and shoulders.
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Imago Pharmaceuticals, Inc. is using nucleic acid therapeutics technology platform for the treatment of spinal muscular atrophy. Nucleic acid therapeutics technology includes large and innovative class of drugs that can modulate the function of target ribonucleic acid, or RNA, to ultimately affect the production of disease-associated proteins. Nucleic acid therapeutics comprise of complex mixtures of various chemical entities known as stereoisomers. Some stereoisomers in these mixtures have therapeutic effects, while others are less beneficial or contribute to undesirable side effects. Uncontrolled stereoisomer drug mixtures can lead to suboptimal efficacy and increased risk and safety concerns.
The research also found that there are several companies that use synthetic sources for the development of drugs for the treatment of spinal muscular atrophy.
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Some of the other key players developing drugs for the treatment of spinal muscular atrophy include Cytokinetics, Inc., F. Hoffman La-Roche Ltd, WAVE Life Sciences Ltd. and others.
Niemann–Pick Disease Therapeutics Pipeline Analysis - Collaboration, and Other Developments
Niemann-Pick Disease is a type of lipid storage disorder characterised by accumulation of fatty substances in organs such as spleen, liver, and brain, eventually causing cell death resulting in malfunctioning of major organ systems. Its pathogenesis involves deficiency of a lysosomal enzyme called acid sphingomyelinase, and is mainly caused by genetic mutation. Niemann-Pick disease is majorly prevalent in North America, South America, Europe, Africa, Asia, and Australia. There are three main types of Niemann-Pick Disease called Type A and B (Acid Sphingomyelinase Deficiency) and Niemann-Pick Disease Type C (NPC). Approximately 1,200 cases of NPA and NPB have been recorded, worldwide, with the majority being Type B or the intermediate form. Cure for Type A disease is yet to be found and researchers are continuously trying new possible treatments including enzyme replacement and gene therapy. For Type D, there is no effective treatment; however, few drugs are available for curing the nervous system symptoms of Type C disease.
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In April 2017, Sucampo Pharmaceuticals acquired Vtesse Inc., including its clinical stage Niemann-Pick disease type C1 drug, VTS-270. However, the Vtesse team will continue the ongoing Phase IIb/III clinical trials for VTS-270. The trials are expected to readout in mid-2018 and the drug could reach the market as early as 2019. In March 2017, CTD Holdings, Inc. began recruitment for a Phase I EU clinical trials of Trappsol® cycloTM to be used for the treatment of Niemann-Pick Disease Type C. In addition to the EU study, CTD Holdings is planning to initiate Phase I clinical trials in the U.S. for evaluating intravenous administration of Trappsol® Cyclo™ in NPC patients. In June 2016, Orphazyme APS announced dosing of two patients with arimoclomol in the Phase 3 ‘AIDNPC’ Niemann-Pick Disease Type C clinical trial programme. For the study, Orphazyme is seeking approval for clinical trial applications with eight national competent authorities and ethics committees.
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The key market players having a therapeutic pipeline for Niemann-Pick disease include CTD Holdings, Inc., Sanofi Genzyme, Alexion Pharmaceuticals, Actelion Pharmaceuticals Ltd, Vtesse Inc., La Jolla Pharmaceutical Company, Merck & Co., Inc., Okklo Life Sciences BV, Orphazyme ApS and Aldagen Inc.
Atrial Fibrillation Therapeutics Pipeline Analysis - Collaboration and Other Developments
Atrial fibrillation therapeutic pipeline is expected to increase on account of increasing prevalence of lifestyle disease such as obesity, diabetes, high blood pressure and cholesterol, which in turn lead to increase in the incidence of the disease. Some other prominent factors driving the pipeline analysis for atrial fibrillation include aging population, growing tobacco and alcohol consumption and rising health awareness among people. According to the recently published data by Centers for Disease Control and Prevention, around 2.7 to 6.1 million people in the U.S. are suffering from atrial fibrillation, with 2% of the infected population being less than 65 years of age. Each year around 750,000 patients get hospitalized because of atrial fibrillation and around 130,000 people die every year. Atrial fibrillation has become one of the major causes of deaths, globally, and costs the United States about $6 billion each year. Annually, medical costs for people who have atrial fibrillation are about $8,705 higher than those who do not have atrial fibrillation. The disease is more prevalent in females as compared to males.
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Atrial fibrillation, also known as AFib, is the condition involving irregular heartbeats resulting in increased risk of ischemia, blood clots, heart failure, stroke and other heart diseases. This condition arises more commonly in people with high blood pressure, heart muscle disease, heart valve disease, heart defect, enlargement of the chambers on the left side of the heart, diabetes, hyperthyroidism, obesity, heavy alcohol and tobacco consumption, smoking and chronic obstructive pulmonary disease. Atrial fibrillation can be occasional, persistent, long-standing persistent and permanent depending on its severity. The symptoms of atrial fibrillation include weakness, breath shortening, chest pain, tiredness, dizziness and palpitations. Symptoms vary from mild to severe in different individuals. Atrial fibrillation is diagnosed by an electrocardiogram, blood tests, chest X-ray and echocardiogram. The treatment of atrial fibrillation includes medications, surgery, pacemaker implant, cardioversion or ablation. The medications usually prescribed work as calcium channel blockers, beta blockers, potassium channel blockers and blood thinners. Atrial fibrillation can be prevented by taking healthy diet, lowering alcohol and caffeine consumption, involving exercise in your daily routine and limiting smoking and tobacco consumption.
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Some of the companies having a pipeline of atrial fibrillation therapeutics include Bristol-Myers Squibb Company, Bayer AG, ARCA Biopharma, Inc., Omeicos Therapeutics GmbH, AstraZeneca plc, Daiichi Sankyo Company Ltd., Gilead Sciences, Inc., Merck & Co., Inc., Allosteros Therapeutics, Inc.
