Von Hippel-Lindau Syndrome Therapeutics - Pipeline Analysis 2020, Clinical Trials & Results

Von Hippel-Lindau syndrome is a genetic disorder characterized by formation of cysts and tumors in different parts of the body. The symptoms associated with the disease depends upon the location of tumor.

However, gait disturbance, dizziness, weakness of the limbs, deafness, high blood pressure, and vision problems are some common symptoms observed in the patients. The available treatment options for the disease are surgical removal of the tumor, high dose irradiation, and chemotherapies.

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Von Hippel-Lindau syndrome is mostly found to be associated with pheochromocytoma. The National Eye Institute (NEI), based in the U.S., is in the process of developing a combination therapy of ranibizumab and E10030 for the treatment of von Hippel-Lindau syndrome.

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The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials.

Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.

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Focal Segmental Glomerulosclerosis (FSGS) Therapeutics - Pipeline Analysis 2020, Clinical Trials & Results

Focal segmental glomerulosclerosis (FSGS) is a glomerular disease, which is characterized by scarring or sclerosis in the kidney. Scarring damages the limited number of glomeruli in the initial stages. It is more common in males than in females, and is very common in African American population.

FSGS can be caused by many diseases like diabetes, sickle cell disease, and HIV infection. It is categorized into two types; primary and secondary FSGS, and can be treated using corticosteroids, ACE inhibitors, immunosuppressive drugs, diuretics, and diet modification.

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Retrophin Inc. is in the process of developing Sparsentan, with dual mechanism of action that combines angiotensin receptor blockade with endothelin receptor blockade for the treatment of FSGS. Some other companies in FSGS pipeline are Variant Pharmaceuticals Inc. and ChemoCentryx Inc.

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The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials.

Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.

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Tic Disorder Therapeutics - Pipeline Analysis 2020, Clinical Trials & Results, Patents, Designations, Collaborations

Tic disorder is a type of mental disorder, characterized by sudden and nonrhythmic movements. According to the World Health Organisation (WHO), this disease can be categorized into five types; transient, combined vocal and multiple motor, unspecified, and chronic motor tic disorder.

Some of the symptoms of the disease are facial grimacing, excessive blinking, uncontrollable movements of the legs, and sound such as throat clearing. Majorly found in children, this disease can be treated by a combination therapy of drugs, that reduce the dopamine concentration in the brain.

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However, cognitive behavioural therapy is also being used for the treatment of this disease. Abide Therapeutics Inc. is in the process of developing ABX-1431 for the treatment of tic disorder. Neurocrine Biosciences is in the process of developing valbenazine as a vesicular monoamine transporter 2 inhibitor for the treatment of this disorder.

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The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials.

Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.

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Spasticity Therapeutics - Pipeline Analysis , Clinical Trials & Results, Patents, Designations, Collaborations

Spasticity is a condition in which certain muscles are continuously contracted. The constant muscle contraction causes stiffness or tightness of the muscles and can interfere with normal movement, speech and gait in a patient.

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Spasticity is usually caused by damage to the portion of the brain or spinal cord that controls voluntary movement. This damage causes a change in the balance of signals between the nervous system and muscles, leading to increased activity in muscles.

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Spasticity negatively affects muscles and joints of the extremities and is particularly harmful to growing children. Currently available treatment options for this disease include oral medications and interventional procedures. Certain conditions that may cause spasticity are traumatic brain injury, spinal cord injury, brain damage due to a lack of oxygen, stroke, encephalitis, and meningitis.

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The drug candidates of for treating this condition include, but are not limited to, Arbaclofen extended release and Botulinum toxin E. Some of the companies having drugs in spasticity pipeline are Osmotica Pharmaceutical, Ipsen Pharma, and Canbex Therapeutics Ltd.

The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials.

Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.

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Polymyalgia Rheumatica Therapeutics - Pipeline Analysis, Clinical Trials & Results

Polymyalgia rheumatica is an inflammatory disorder which causes stiffness and aching, and usually affects adults over the age of 50 years. This disease affects women somewhat more than men. The cause of polymyalgia rheumatica is unknown.

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Polymyalgia rheumatica is related to another inflammatory disorder, giant cell arteritis, which can cause vision difficulties, scalp tenderness, jaw pain, and headaches. Pain in shoulders, neck, upper arms, buttocks, hips and thighs; stiffness in the affected areas; limited range of motion in affected areas; and stiffness in wrists, elbows and knees are some of the common symptoms of polymyalgia rheumatica.

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Involvement of the upper arms, with trouble raising them above the shoulders is common in the patients with this disease. Corticosteroids and nonsteroidal anti-inflammatory drugs (NSAIDs) are common medications for the treatment of polymyalgia rheumatica. GlaxoSmithKline plc is in the process of developing sirukumab as an interleukin 6 (IL6) human monoclonal antibody for the treatment of polymyalgia rheumatica. Other than this Chugai Pharmaceutical Co. Ltd., and Eli Lilly and Company are also involved in polymyalgia rheumatica pipeline.

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The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials.

Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.

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Tagraxofusp - API Insight by Pipeline Analysis, Clinical Trials & Results, Patents, Designations, Collaborations, and Other Developments

Tagraxofusp, sold under the brand name of Elzonris, is an Interleukin 3 (IL-3) conjugated truncated diphtheria toxin. It is composed of catalytic and translocation domains of diphtheria toxin fused via Met-His linker to a full-length human IL-3. Tagraxofusp was developed by Stemline Therapeutics Inc. and was approved in December 2018, as the first therapy for blastic plasmacytoid dendritic cell neoplasm. This drug achieved approval after being designed with the title of breakthrough therapy, priority review, and orphan drug status.

Tagraxofusp is indicated for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and pediatric patients over 2 years old. This treatment allows an alternative for the previous intense treatment which consisted of intensive chemotherapy followed by bone marrow transplantation. Some of the side-effects include fatigue, peripheral edema and weight increase.

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This report provides details of the drug and the API manufacturers across the 7 major markets which includes U.S, EU5, and Japan. It includes the overview, mechanism, regulatory milestones, strategic developments, the historical and forecasted sales. 

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The pipeline analysis of the drug by phase which would include the product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology and others.

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Androgenetic Alopecia Therapeutics Pipeline Analysis by Stages, Drug Class, Company Profile, Phase of Development, Clinical Trial & Discovery Stage Insights

Androgenetic Alopecia Therapeutics – Competitive Landscape, Epidemiology Forecast, and Pipeline Analysis, 2019 published by Pharma Proff, androgenetic alopecia therapeutics currently exhibits a proliferating pipeline with 15+ therapeutic candidates.

Androgenetic Alopecia Therapeutics Pipeline Insights

Androgenetic alopecia is a genetic disorder of patterned hair loss that affects both men and women. The disorder shortens hair growth cycle due to excess of androgen, which eventually delays hair growth. Some of the major symptoms include gradual recession of frontal hairline, increased hair fall, bitemporal recession of hair, and thinning of the hair. The symptoms start appearing at the age of 20 and are seen to be prominent in people aged 40 years and above. The disorder leads to hair thinning near crown region, and maybe caused due to various genetic as well as environmental factors. The disorder can also occur due to hormonal imbalance, improper functioning of androgen hormones, poor diet, and excessive smoking.

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Insights into Pipeline Segments

According to the research, many drugs being developed for androgenetic alopecia, which are administered topically. It has been found that topical route of administration is easy to use, non-invasive, and ensures high level of patient satisfaction.

Positive Clinical Trial Results are Expected to Drive Androgenetic Alopecia Therapeutics Pipeline

There are several companies which have shown positive results of clinical trials. For instance, in February 2019, Cassiopea SpA announced effective result of Phase II clinical trial of Breezula for treatment of androgenetic alopecia.

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Technological Advancements Play a Pivotal Role in Androgenetic Alopecia Therapeutics Development

It has been observed that many pharmaceutical companies are developing new and improved technologies for the development of androgenetic alopecia therapeutics. These technologies play an important role in the drug development by helping the companies to develop more specific and effective targeted therapies. For instance, RepliCel Life Sciences is using its proprietary cell therapy technology to develop therapeutic candidates for pattern baldness and other disorders. It utilizes dermal sheath cup cells, which induce the growth of cells in the area of baldness.

Samumed LLC, Brickell Biotech Inc., Aclaris Therapeutics Inc., Cassiopea S.p.A, Kerastem Technologies LLC, Almirall S.A., and RepliCel Life Sciences are the major players involved in the development of drug candidates for the treatment of androgenetic alopecia.

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Adrenoleukodystrophy (ALD) Therapeutics Pipeline Analysis by Therapeutics, Diagnostics, Patient, Drugs Policy and Regulatory Landscape

Adrenoleukodystrophy (ALD) Therapeutics Pipeline Analysis 2019, Clinical Trials and Results, Patents, Designations, Collaborations, and Other Developments published by Pharma Proff, ALD currently exhibits a proliferating pipeline with 10 therapeutic candidates.

ALD Pipeline Insights

ALD is a X-linked genetic disease which falls under the family of leukodystrophies disorder. The disease is monogenetically inherited from parents to offspring, with 100% penetrance in men and 65% in heterozygous women. It is a multi-system disorder which prominently affects both central and peripheral nervous system, resulting in blindness, seizures and hyperactivity.

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The pathophysiologic condition of disease includes the accumulation of saturated very long chain fatty acids (VLCFA) in serum and tissues of central nervous system, which is a major factor for immune system abnormality and demyelination of neurons. It mainly affects nervous system and adrenal glands and causes various adrenal and neurological problems, such as leg stiffness, muscles spasms and weakness, urinary problems, and sexual dysfunction.

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Insights into Pipeline Segments

According to the research, many drugs acting as a therapeutic against ALD are administered orally. It has been found that oral route of administration is easy to use, non-invasive, and ensures high level of patient satisfaction.

It has been observed that regulatory bodies are granting designations to the drugs in ALD pipeline, in order to pace up the development process. For instance, bluebird bio Inc. ‘s drug candidate, Lenti-D, received Orphan Drug Designation by the USFDA and EMA in 2012; and Breakthrough Therapy Designation by the USFDA in 2018. Similarly, Minoryx Therapeutics S.L. drug, MIN-102, received Orphan Drug Designation by the USFDA in 2017.

Some of the key players involved in the development of ALD are Poxel SA, bluebird bio Inc., Minoryx Therapeutics SL, NeuroVia Inc., SOM Innovation Biotech SL, Viking Therapeutics Inc., Orpheris Inc., MedDay Pharmaceuticals, Applied Genetic Technologies Corporation (AGTC), and Neuralgene.

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Musculoskeletal Pain Therapeutics - Pipeline Analysis , Clinical Trials & Results, Patents, Designations

Musculoskeletal pain is a known consequence of repetitive strain, overuse, and work-related musculoskeletal disorders. These injuries include a variety of disorders that cause pain in muscles, bones, joints, or surrounding structures. This pain can be acute or chronic, diffuse or focal. Low back pain is the most common example of chronic musculoskeletal pain. Other musculoskeletal pain includes tendonitis and tendinosis, myalgia, neuropathies, and stress fractures.

                                                 

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The pathophysiology of musculoskeletal pain is not completely clear, but inflammation, fibrosis, tissue degradation, neurotransmitters, and neurosensory disturbances have been implicated. Some clinical symptoms of musculoskeletal pain include local symptoms of pain or widespread and persistent pain, tenderness, peripheral nerve irritation, weakness, and limited motion and stiffness.

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The drug candidates in musculoskeletal pain pipeline include, but are not limited to, NEO 6860, Gefapixant, and Cebranopadol. Neomed, Merck & Co. and Grunenthal Group are some companies involved in musculoskeletal pain pipeline.

The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials.

Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.

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Intermittent Claudication Therapeutics - Pipeline Analysis, Clinical Trials & Results

Intermittent claudication refers to aching or squeezing pain in thighs, buttocks or feet during exercise, due to insufficient blood circulation resulting from peripheral arterial disease. The probability of developing peripheral arterial disease increases with the age.

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Smoking, high blood pressure, atherosclerosis, diabetes, and high cholesterol are some of the risk factors associated with the development of peripheral arterial disease, resulting in intermittent claudication. Intermittent claudication is detected by the pulse and blood pressure in legs and by arteriogram. The standard treatments available for intermittent claudication are angioplasty and surgery.

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 Pluristem Ltd. is in the process of developing PLX-PAD for the treatment of intermittent claudication associated with peripheral arterial disease. Some of the companies involved in intermittent claudication pipeline include Kowa Company Ltd., and Nissan Chemical Industries Ltd. Among others.

The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials.

Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.

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Angioimmunoblastic T-Cell Lymphoma (AITL) Therapeutics - Pipeline Analysis, Clinical Trials & Results, Patents, Designations

Angioimmunoblastic T-cell lymphoma is a rare type of peripheral T-cell lymphoma which is characterized by lymphadenopathy, fever, and arthritis. The signs and symptoms associated with the disease include pleural effusion, arthritis, edema, ascites and pruritic skin rash. The neoplastic cells related to the disease are found in the liver, spleen, lymph nodes and bone marrow.

Angioimmunoblastic T-cell lymphoma can be treated with multiagent chemotherapy regimen. In addition, steroid is used to get relieve from the symptoms in reaction to inflammation. The National Cancer Institute is in the process of developing MEDI-570 as an inducible T-cell co-stimulator protein antagonist for the treatment of angioimmunoblastic T-cell lymphoma.

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The Lymphoma Academic Research Organisation is also in the process of developing lenalidomide for the treatment of this medical condition. Some of the other companies, universities, and organizations having drugs for angioimmunoblastic T-cell lymphoma in their pipeline includes The Lymphoma Academic Research Organization, PharmaMar S.A., Innate Pharma S.A. and others.

The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials.

Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.

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Fibrodysplasia Ossificans Progressiva (FOP) Therapeutics - Pipeline Analysis , Clinical Trials & Results

Fibrodysplasia ossificans progressiva (FOP), also known as myositis ossificans progressive, is a rare inherited connective tissue disorder characterized by abnormal bone development in ligaments, tendons, and skeletal muscles.

Precisely, FOP causes the body's skeletal muscles and soft connective tissues to undergo a metamorphosis, transforming into bone, gradually locking joints and making movement difficult. FOP is inherited as an autosomal dominant trait with complete penetrance. Standard therapies available for FOP are; corticosteroids and non-steroidal anti-inflammatory medication for pain and swelling associated with FOP; occupational therapy; and genetic counselling.

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Regeneron Pharmaceuticals Inc. is in the process of developing REGN2477 as an activin A antibody which act as an activin modulator for the treatment of FOP. Some of the companies in FOP pipeline are Clementia Pharmaceuticals Inc. and Regeneron Pharmaceuticals Inc. among others.

The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials.

Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.

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Hypopharyngeal Cancer Therapeutics - Pipeline Analysis, Clinical Trials & Results, Patents, Designations

Hypopharyngeal cancer, a type of head and neck cancer, develops in hypopharynx. Pharynx is a hollow tube about five inches long, and the bottom part of the pharynx called hypopharynx. Most of the hypopharyngeal malignancy occurs in squamous cells.

Some of the major risk factors associated with the disease are smoking, heavy drinking, tobacco consumption, Plummer-Vinson syndrome, and intake of nutrient deficit diet. The signs and symptoms of the disease are ear pain, voice change, sore throat, difficulty in swallowing, and lump in the neck.

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Biotech Pharmaceutical Co. Ltd. (BPL) is in the process of developing nimotuzumab as an epidermal growth factor receptor antagonist for the treatment of hypopharyngeal cancer. Novartis AG, and the Stanford University are also involved in development of drugs for hypopharyngeal cancer.

The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials.

Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.

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Visceral Pain Therapeutics - Pipeline Analysis , Clinical Trials & Results, Patents, Designations, Collaborations

Visceral pain is the most frequent form of clinically relevant pain for those patients seek medical attention. There are several causes of visceral pain. Nociceptive pain causes direct injury of an internal organs, that leads to cardiac ischemic, and peptic ulcer. Medications for visceral pain treatment includes analgesics such as nonsteroidal anti-inflammatory drugs, paracetamol and serotonergic compounds.

Opioids are also used for the treatment of a range of acute to chronic visceral pains. Sacral nerve stimulation for interstitial cystitis and psychological management are some non-pharmacological treatment available for the management of visceral pain.

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The drug candidates in visceral pain therapeutics pipeline include, but not limited to, NEO5937, ANAVEX 1066 and APD371. Neomed Management AS, Arena Pharmaceuticals Inc. and Anavex Life Sciences Corp. are some of the major companies having drugs in the visceral pain therapeutic pipeline. 

The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials.

Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.

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Tay-Sachs Disease Therapeutics - Pipeline Analysis , Clinical Trials & Results, Patents, Designations

Tay-Sachs is a lysosomal storage disorder caused by the deficiency of vital enzymes called beta-hexosaminidase A (Hex-A). The role of Hex-A is to degrade a fatty substance or lipid called GM-2 ganglioside. In the absence of the Hex-A enzymes, GM-2 accumulates abnormally in cells, especially in the nerve cells or neurons, of the brain.

In children, the destructive process begins in the fetus early in pregnancy. However, a baby with Tay-Sachs disease appears normal till six months of age when its develops slowly. By about two years of age, most children experience recurrent seizures and diminishing mental functioning. The infant gradually regresses, and is eventually unable to crawl, turn over, sit or reach out. There is no specific treatment for Tay-Sachs disease.

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The drug candidates in Tay-Sachs therapeutics pipeline include, but not limited to, CCP-010. Some of the major companies having drugs in the Tay-Sachs therapeutics pipeline includes Lysogene, and Castle Creek Pharmaceuticals.

The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials.

Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.

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