Chronic Myelocytic Leukemia Therapeutics Pipeline Analysis - Clinical Trials & Results, Patent and Developments

Chronicmyelocytic leukemia therapeutic pipeline analysis is expected to grow rapidly due to the increasing prevalence of the disease, globally. Some of the main factors driving the pipeline analysis for chronic myelocytic leukemia include increasing aging population and rising prevalence of chronic myelocytic leukemia, and increasing health awareness. Chronic myelocytic leukemia have high incidence rate in men and is the second most common type of cancer occurring in male adults. Women, above 50 years of age are at high risk to develop chronic myelocytic leukemia.

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Chronic myeloid leukemia is the tumour that occurs in blood cells and bone marrow, which is the soft parts inside bones where blood cells are produced. Chronic myelocytic leukemia usually occurs in middle-aged or older adults. It occurs due to the swapping of DNA between chromosome 9 nine and chromosome 22. The resultant abnormal chromosome is known as Philadelphia chromosome. This swapping forms a new gene, BCR-ABL which is the type of protein, tyrosine kinase. This protein causes chronic myelocytic leukemia cells to grow and reproduce uncontrollable manner. One of the main cause of chronic myelocytic leukemia is exposure to high radiation. The symptoms of chronic myelocytic leukemia include stroke, dizziness, change in vision, swollen glands, high fever, bruises, weight loss, pain in bones, short of breath and night sweats. Chronic myeloid leukemia can be diagnosed by performing a bone marrow test, complete blood count, FISH test and ultrasound. The treatment for chronic myeloid leukemia includes drug therapy and stem cell transplant.

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Some of the companies having a pipeline of chronic myelocytic leukemia therapeutics include Bristol Myers Squibb Company, ARIAD Pharmaceuticals, Inc., Novartis AG, Pfizer Inc., Teva Pharmaceutical Industries Ltd., Hospira, Prism Pharma, Incyte Corp., Otsuka Pharmaceutical Co., Ltd., Bio-Path Holdings, Hybrigenics Corporation, Fusion Pharma LLC.

Fabry Disease Therapeutics Pipeline Analysis 2017

Fabry disease is a type of inherited genetic disease caused due to abnormal deposition of a fatty substance, called globotriaosylcera-mide, in blood vessel walls throughout the body. This further leads to development of other complications, which result in severe morbidities and can also be fatal, in some cases. Anticipated growth of therapeutic pipeline of fabry disease can be attributed to the rareness of the disease, as it is usually found in males at an estimated ratio of one in 40,000 to 60,000 males. Although, the disorder occurs in females as well, its prevalence is still unknown. This disease can be cured using gene therapy and cell therapy, which are still in the development phase. With increasing support from various funding agencies such as National Organization of Rare Disorders (NORD), new and innovative therapies are being developed to cure the problem. Therefore, the therapeutic pipeline of Fabry disease is increasing.

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In November 2016, FGK Clinical Research GmbH0 started Phase I, open-label, multi-center study to evaluate pharmacokinetics, pharmacodynamics, and safety of Moss-aGal in patients suffering from fabry disease. This study was completed in February 2017. Moss-aGal is a recombinant human alpha-galactosidase that is produced in moss. It is expected to be the first of its kind enzyme replacement therapy (ERT) for patients with the genetic lysosomal storage disorder, which exhibits optimized N-glycosylation patterns of the protein alpha-galactosidase A. In July 2016, Ozmosis Research Inc. started a Phase I clinical study on autologous stem cell transplantation of cluster of 34 positive (CD34+) cells engineered to express Alpha-galactosidase-A in patients with fabry disease. This study is expected to be completed in July 2022. In June 2016, Protalix Biotherapeutics Inc., started a Phase III trial for testing safety and efficacy of PRX-102, as compared to Agalsidase beta on renal function in patients with fabry disease previously treated with Agalsidase beta. PRX 102 demonstrated improved circulatory half-life, with better enzyme activity in target organs affected by fabry disease.

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Some of the companies having a pipeline of fabry disease therapeutics include Greenovation Biotech GmbH, GlaxoSmithKline Plc, Protalix Biotherapeutics, Inc., Amicus Therapeutics, Inc, Shire Plc, Genzyme Corporation, Ozmosis Research Inc.

Myasthenia Gravis Therapeutics Pipeline Analysis 2017 - Clinical Trials & Results and Developments

Myasthenia gravis is a neuromuscular disease that leads to varying degrees of skeletal muscle weakness. It is an autoimmune disease resulting from the antibodies blocking or destroying nicotinic acetylcholine receptors at the junction between the nerve and muscle, thus preventing nerve impulses rom triggering muscle contractions. Growth is anticipated in the myasthenia gravis therapeuticspipeline on account of increasing participation of organizations, such as National Organization for Rare Disorder, that support research and development activities in field. The unbearable complications such as difficulty in vision, swallowing, weakness in upper arms and legs and impaired speech are also contributing to the growth of pipeline therapeutics of myasthenia gravis.

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In August 2005, Debiopharm S.A. entered into a collaboration with DeveloGen AG for development of PTR-262, a novel peptide drug for the treatment of myasthenia gravis (MG). PTR-262 is derived from the myasthenogenic epitopes of the acetylcholine receptor (AChR) alpha-subunit, which specifically arrests the autoimmune destruction of AChR. Under the terms of the agreement, DeveloGen AG would receive milestone payments and, upon commercialisation of the product, royalties based on revenues of Debiopharm S.A., from worldwide sales. Debiopharm S.A. is an independent company, specialising in oncology, endocrinology and niche products, whereas, DeveloGen AG is a biopharmaceutical company, which mainly focuses on development of novel treatments for metabolic disorders. In March, 2016, Penn centre for innovation had developed therapeutic vaccine for myasthenia gravis. The advantages of vaccine are long lasting benefits, potentially permanent protection, prevention as well as reversal of chronic myasthenia gravis, potent safe and rapid acting.

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Some of the companies having a pipeline of myasthenia gravis therapeutics include UCB Pharma, Ltd. Novartis AG, Grifols Therapeutics Inc., Bristol-Myers Squibb Company, Catalyst Pharmaceuticals, Inc., DeveloGen AG, Debiopharm S.A.

Chronic Kidney Disease Therapeutics Pipeline Analysis 2017 - Designation, Collaboration, and Other Developments

The therapeutics pipeline of chronic kidney disease has been increasing on account of rising prevalence of diabetes and high blood pressure in patients. The diabetes and blood pressure are the main causes of chronic kidney disease, which is also related to cardiovascular disease. Due to altered lifestyle, the chances of incidence of high blood pressure and diabetes is on a rise. Currently, the prevalence of chronic kidney disease is around 14% in Australia, as studied by Oxford Academic Journal of Nephrology Dialysis Transplantation. According to, National Institute of Diabetes and Digestive and Kidney Disease, the number of kidney failure cases among the Americans stood at more than 661,000, out of which 468,000 patients are undergoing dialysis and 193,000 patients are living with functional kidney transplant. The increasing awareness of healthcare issues also contributes towards the of development of therapeutics pipeline for chronic kidney disease.

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Chronic kidney disease is the condition that can lead to the damage of the kidneys. Some of the main symptoms of chronic kidney disease are, weight loss, insomnia, tiredness, blood in urine, feeling sick, muscle cramps shortness of breath.

Many companies are conducting clinical trials for the development of therapeutics for chronic kidney disease. Astellas Pharma, Inc., is in the process of introducing a drug by the name ASP1517, which is in Phase III stage of its clinical development. ASP1517 is an orally administered small molecule which is used for the treatment of anaemia associated with chronic kidney disease. AstraZeneca Plc is also introducing a drug named, Dapagliflozin, which is currently in its Phase III stage of clnical development. Dapagliflozin is a sodium-glucose cotransporter II inhibitor.

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Some of the companies having a pipeline of chronic kidney disease therapeutics include Astellas Pharma Inc., AstraZeneca Plc, Proteon Therapeutics, Inc., RegenMed (Cayman) Ltd, Gilead Sciences, Inc., GlaxoSmithKline Plc, Relypsa, Inc., Chugai Pharmaceutical, Inc., Kissei Pharmaceutical Co., Ltd, Boryung Pharmaceutical Co., Ltd.

Cushing's Syndrome Therapeutics Pipeline Analysis 2017 - Collaboration and Other Developments

The expending growth of pipeline for Cushing’s syndrome therapeutics is attributed to the increase in prevalence of inflammatory disorders. For the treatment of various inflammatory disorders patients use corticosteroids, the long-term use of corticosteroids can lead to Cushing’s syndrome. In many diseases such as asthma and rheumatoid arthritis, corticosteroids are being used, for a lifelong period but at low doses. However, when the disease fails to respond to lower doses of corticosteroids, the dose needs to be increased. The pipeline for Cushing’s syndrome is majorly driven by factors such as, increasing healthcare awareness in patients and the occurrence of abnormal growth of cells in pituitary gland which can further lead to the formation of tumour.

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Cushing’s syndrome is a condition that occurs when a person is exposed to the hormone cortisol at a very high level, for a longer period of time, this is sometimes called as hypercortisolism. It mostly affects obese people at the aged between 20 to 50 years, who have type 2 diabetes and high blood pressure. The symptoms of the Cushing’s syndrome, include, round face, upper body obesity, deposition of fat around neck and relatively lean arms and legs. In some cases, skin of the patients become fragile, heals poorly, bruises easily and purple or pink marks appear on the abdomen, buttocks, thigh and breast. Bones become weakened and spinal and column fracture can occur when person is doing any routine activity like bending, lifting etc. Other symptoms include severe fatigue, increased thirst and urination, irritability, anxiety, depression and weak muscles.

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In April 2012, Strongbridge Biopharma Plc was in the process of collaboration with Moulder Center Drug Discovery for the development of new therapies of cortisol modulation for Cushing syndrome therapeutics. In July 2014, Bristol-Myers Squibb Company and Ono Pharmaceutical Co., Ltd. announced that the companies are undergoing a strategic immuno-oncology collaboration in Japan, South Korea and Taiwan, which includes the development and commercialization of immune mediated endocrinopathies. In September 2008, Corcept Therapeutics and Eli Lilly and Company announced that the companies would continue the collaboration for testing the effectiveness of GRII receptor antagonist in rat models of olanzapine induced weight gain.

Some of the companies having a pipeline of Cushing’s syndrome therapeutics include Corcept Therapeutics, Millendo Therapeutics, Inc., Strongbridge Biopharma Plc, Novartis AG. Ono Pharmaceutical Co., Ltd. Bristol-Myers Squibb Company.

Psoriatic Arthritis Therapeutics Pipeline Analysis - Clinical Trials & Results, Collaboration and Other Developments

The number of drug candidates in pipeline for psoriatic arthritis are expected to increase on account of increase in the prevalence of psoriasis, globally. Psoriasis is the skin disease which is more common in Caucasian as compared to the African American and Asian Americans. The disease is symptomatized by white and scaly red patches. The prevalence of psoriatic arthritis has been increasing mainly in the patients, who are more susceptible to infection that may activate their immune system. Increase in mortality rate, other inflammatory disease complication and awareness regarding health issues are the main reasons, that are responsible for the growth of psoriatic arthritis therapeutics pipeline. According to the data published by National Institute of Health in 2014, the incidence of psoriatic arthritis ranged from 0.1 out of 100000 people in Japan to 23.1 out of 100000 people in Finland, in the Europe and America the prevalence of psoriatic arthritis varied from 0.02-0.42% of the total population. Indians have highest prevalence of psoriatic arthritis in, Singapore’s multi-ethnic population.

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Psoriatic arthritis is a chronic inflammatory disease which involves inflammation of skin, synovial tissue and usually gives seronegative results for rheumatoid factor. Other complex diseases which are associated with psoriatic arthritis include ankylosing spondylitis, arthritis associated with inflammatory bowel disease and reactive arthritis. The psoriatic arthritis is diagnoses is done on the basis of the medical or family history and physical examinations such as magnetic field resonance imaging and x-ray of the affected joints. Currently, researchers are looking for better ways to diagnose psoriatic arthritis accurately, since at times a lot of similarity exist between psoriatic arthritis and other inflammatory form of arthritis. Contrast-enhanced MRI has also been developed by the researchers that can differentiate between psoriatic arthritis and rheumatoid arthritis in hand and wrist.

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Some of the companies having a pipeline of Psoriatic arthritis therapeutics include Novartis AG, Amgen Plc, Galapagos NV, AbbVie, Inc., Pfizer, Inc., Celgene Corporation, Janssen Pharmaceutical K.K.

Juvenile idiopathic arthritis Therapeutics Pipeline Analysis 2017 - Clinical Trials & Results, Collaboration and Other Developments

The expanding pipeline for juvenile idiopathic arthritis can be attributed to the increase in its prevalence. One of the major factor that stimulate the growth of pipeline and research is that the cause of juvenile idiopathic arthritis is not known. The juvenile idiopathic arthritis occurs mostly in children before 16 years of age. Since the disease occurs only in children, so the safety and efficacy concerns of the drugs is given preference so that it can be treated without any side effects. Some of the other factors driving the pipeline growth include, raising awareness of health issues among children and less number of drugs available for therapy against juvenile idiopathic arthritis. Currently, one in 1,000 child in the U.S. suffers with juvenile idiopathic arthritis. Females are more susceptible to juvenile idiopathic arthritis than males.

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Juvenile idiopathic arthritis can be symptomatized as swelling, joint pain, tenderness, stiffness and fever that last for approximately more than 6 weeks. Juvenile idiopathic arthritis, mostly occurs when body’s immune system erroneously attacks its own healthy tissues and cells, which result in inflammation, pain, heat, redness and swelling. Researchers have studied that, either environmental factors or genetic tendency of child act as triggers for the occurrence of the disease. Juvenile idiopathic arthritis affects mostly joints in knees, hand and feet. Sometimes, severe complication can occur in children with juvenile idiopathic arthritis such as eye inflammation. Currently, no medical test is approved for diagnosis of juvenile idiopathic arthritis. Physicians diagnose juvenile idiopathic arthritis by examining the medical history of the patient and the result of other tests that help in ruling out other morbidities.

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Some of the companies having a pipeline of juvenile idiopathic arthritis therapeutics include, Regeneron Pharmaceuticals, Inc., F. Hoffmann-La Roche AG, Bristol-Myers Squibb Company, Pfizer, Inc., Novartis AG.