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Spinal Muscular Atrophy Therapeutics Pipeline Analysis 2017

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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

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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.

Cardiovascular Information System Market Analysis, Size, Growth and Forecast to 2023

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The global cardiovascular information system market is witnessing significant growth due to increasing prevalence of cardiovascular diseases, growing government initiates towards electronic health record system implementation and growing rate of medication errors. The remote access to data and images, and reduced IT burden in the diagnostic center and hospitals are supporting the demand of CVIS. Moreover, better interoperability and increasing healthcare cost are further expected to boost the market. Based on mode of operation, web-based segment leads the global market in terms of size, owing to high rate of awareness, improved interoperability, reduced IT burden in hospitals and relatively less cost. Based on component, the market for service segment is expected to grow at the fastest rate during the forecast period, due to upgradation of services for the complex software introduction and requirement for extensive training.

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The increasing demand for cloud based solutions is expected to create opportunities for the players in the market. The unexplored market in developing economies is also creating abundant opportunities for the cardiovascular information system market to grow at a significant rate in the coming years. However, high cost of cardiovascular information, lack of standard healthcare protocol towards implementation of CVIS solution in developing countries and reluctance of healthcare provides to implement cardiovascular information system are the key factors hampering the growth of the global market.

Cardiovascular disease is the disease of heart and blood vessels, which includes conditions such as hypertension, coronary heart disease, heart failure, cerebrovascular disease and rheumatic heart disease. It is the leading cause of premature death around the world. According to the WHO, globally about 17.5 million people died due to cardiovascular diseases in 2012, while the number is expected to reach 23.3 million by 2030.

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The key players operating in the global cardiovascular information system market are Philips Healthcare, McKesson Corporation, Philips Healthcare, General Electric Company, Siemens AG, AGFA Healthcare and Cerner Corporation.

Alzheimer’s Disease Pipeline Analysis, 2017 - Clinical Trials & Results and Other Developments

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The study analyzed that the Alzheimer’s disease pipeline comprises 89 drug candidates in different stages of development.

As per the findings of the research, majority of the active drug candidates of Alzheimer’s disease pipeline are being developed to be administered by oral route.

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AC Immune, Ltd. is using SupraAntigen technology platform for the development of Crenezumab, ACI-24 and ACI-35 for Alzheimer's disease. This technology is used to produce conformation specific antibodies and is used to create products for active immunization (vaccines) and passive immunization (antibodies).

The research finds that different companies have collaborated for the development of Alzheimer’s disease pipeline. In March 2014, Eisai Co., Ltd. and Biogen Idec collaborated to develop and commercialize two of Eisai's clinical candidates, E2609 and BAN2401, for Alzheimer’s disease.

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Some of the key players developing drugs for Alzheimer’s disease are Eli Lilly and Company, Genentech, Inc., Eisai Co., Ltd., and others.

Oral Mucositis Therapeutics Pipeline Analysis, 2017 - Collaboration and Other Developments

The study analyzed that the oral mucositis therapeutics pipeline comprises approximately 18 drug candidates in different stages of development. Oral mucositis is the most common complication of chemotherapy in cancer patients. It causes damage to mucosal lining of the mouth which results in formation of ulcers. It can result in several problems that includes severe pain in mouth, nutritional problems as a result of inability to eat and increased risk of infection due to open sores in the mucosa.

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Oragenics, Inc. is using ActoBiotics technology platform for the development of their drug candidates for the treatment of oral mucositis. The technology platform produces a new class of orally delivered biopharmaceuticals. Through ActoBiotics technology platform, food-grade microbes are engineered to generate biologically contained ActoBiotics therapeutics for in situ expression and secretion of novel proteins and peptides including cytokines, enzymes, hormones and monoclonal antibodies within the body.

The research also found that various companies received designations from the USFDA for the development of the drugs in the oral mucositis therapeutics pipeline. In November 2016, USFDA granted Fast Track designation to AG013 for the treatment of oral mucositis.

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Some of the other key players developing drugs for the treatment of oral mucositis include Daewoong Pharmaceutical Co., Ltd., Cellceutix Corporation, Soligenix, Inc. and others.

Restless Legs Syndrome Market Analysis, Size, Growth and Forecast to 2023

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Restless legs syndrome or RLS is a neurological disease. RLS is also called as Willis-Ekbom disease. Patients suffering from RLS disease have an irresistible desire to move their feet. The continual movement of the legs leads to pain in the limbs, which leads to the unusual sleep pattern of patients.

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Restless legs syndrome increases at night when the patient is at rest. RLS are common in both, male and female; however the disease has its highest impact on woman who are elderly or in some cases woman with the age 35 to 50 years. Some of the most common symptoms of the restless legs syndrome are continue itching and pain in the legs, and unusual sleep pattern. The restless legs syndrome affects the quality of life of the person suffering from the disease.

The exact reason behind the restless legs syndrome is not known precisely, however, it is believed the disease is caused due to genetic factors or due to iron deficiency in the brain or due to the disorders in dopaminergic balance.

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The exact treatment of this disease is not present till now. However the available drug in the market helps in the indicative treatment of the disease. Some of the drug in the restless legs syndrome market such as sulfonamide by safinamide by Newron Pharmaceuticals reduces the symptoms by novel action such as potent, reversible and selective inhibition of MAO-B, without disturbing the MAO-A and dopamine uptake. This enhances the glutamate release and dopaminergic function of the patients; it also reduces the dopamine degradation. One other drug: SER-214 manufacture by Serina is used widely in the restless legs syndrome treatment; it uses a water-soluble polymer poly (2-alkyloxazoline)-based drug delivery technology this drug can be combined with the polymer and transported to the patients.

Some of the major players operating in the global restless legs syndrome market include, GlaxoSmithKline plc., Ligand Pharmaceuticals Incorporated, axxonis Pharma AG, Kyowa Hakko Kirin Co., Jazz Pharmaceuticals, Inc., Omeros Corporation, Manhattan Pharmaceuticals, Boehringer Ingelheim GmbH, UCB SA.