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NewAlpha-1 Antitrypsin Deficiency (A1AD) Clinical Trial Analysis by Trial Phase, Trial Status, Trial Counts, End Points, Status, Sponsor Type and Top Countries, 2023 Update
GlobalData's clinical trial report, “Alpha-1 Antitrypsin Deficiency (A1AD) Clinical Trial Analysis by Trial Phase, Trial Status, Trial Counts, End Points, Status, Sponsor Type and Top Countries, 2023 Update" provides an overview of Alpha-1 Antitrypsin Deficiency (A1AD) Clinical trials scenario. This report provides top line data relating to the clinical trials on Alpha-1 Antitrypsin Deficiency (A1AD). Report includes an overview of trial numbers and their average enrollment in top countries conducted across the globe. The report offers coverage of disease clinical...
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NewDuchenne Muscular Dystrophy Clinical Trial Analysis by Trial Phase, Trial Status, Trial Counts, End Points, Status, Sponsor Type and Top Countries, 2023 Update
Duchenne Muscular Dystrophy Clinical Trials Market Report Overview The Duchenne Muscular Dystrophy clinical trial market research report provides an overview of the Duchenne Muscular Dystrophy clinical trials scenario. The report provides top-line data relating to the clinical trials on Duchenne Muscular Dystrophy. It also includes an overview of trial numbers and their average enrollment in top countries conducted across the globe. Moreover, it offers coverage of disease clinical trials by region, country (G7 & E7), phase, trial status, end points...
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NewMucopolysaccharidosis I (MPS I) (Hurler Syndrome) Clinical Trial Analysis by Trial Phase, Trial Status, Trial Counts, End Points, Status, Sponsor Type and Top Countries, 2023 Update
Mucopolysaccharidosis I Clinical Trials Market Report Overview The Mucopolysaccharidosis I clinical trial market research report provides an overview of the Mucopolysaccharidosis I clinical trials scenario. The report provides top-line data relating to the clinical trials on Mucopolysaccharidosis I. It also includes an overview of trial numbers and their average enrollment in top countries conducted across the globe. Moreover, it offers coverage of disease clinical trials by region, country (G7 & E7), phase, trial status, end points status, and sponsor type....
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Sector Analysis
Mucopolysaccharidosis II (MPS II) Marketed and Pipeline Drugs Assessment, Clinical Trials and Competitive Landscape
Mucopolysaccharidosis II Marketed and Pipeline Drugs Report Overview Mucopolysaccharidosis II (MPS II) is an inherited lysosomal storage disorder of carbohydrate metabolism that occurs almost exclusively in males. In patients with MPS II, glycosaminoglycans accumulate within tissues and organs, contributing to the signs and symptoms of the disease. MPS II affects multiple organs and physiologic systems. It is a progressively debilitating disorder; however, the rate of progression varies among affected individuals. MPS II is caused by genetic changes in the iduronate...
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Prader-Willi Syndrome (PWS) Clinical Trial Analysis by Trial Phase, Trial Status, Trial Counts, End Points, Status, Sponsor Type and Top Countries, 2023 Update
Prader-Willi Syndrome (PWS) Clinical Trials Market Report Overview The Prader-Willi Syndrome (PWS) clinical trial market research report provides an overview of the PWS clinical trials scenario. The report provides top-line data relating to the clinical trials on PWS. It also includes an overview of trial numbers and their average enrollment in top countries conducted across the globe. Moreover, it offers coverage of disease clinical trials by region, country (G7 & E7), phase, trial status, end points status, and sponsor type.
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Sector Analysis
Angelman Syndrome (AS) Marketed and Pipeline Drugs Assessment, Clinical Trials and Competitive Landscape
Angelman Syndrome Disorder Marketed and Pipeline Drugs Report Overview Angelman Syndrome (AS) is a genetic condition that affects the nervous system and causes severe physical disabilities, learning disabilities, motor dysfunction, seizure disorder, and often a happy, sociable disposition. A person with AS will have a near-normal life expectancy, but they will need support throughout their life. Some delayed development can be observed at ages 6–12 months, while other common symptoms usually occur before age three years. The Angelman Syndrome marketed...
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Fragile X Syndrome Clinical Trial Analysis by Trial Phase, Trial Status, Trial Counts, End Points, Status, Sponsor Type and Top Countries, 2023 Update
Fragile X Syndrome Clinical Trials Market Report Overview The Fragile X Syndrome clinical trial market research report provides an overview of the Fragile X Syndrome clinical trials scenario. The report provides top-line data relating to the clinical trials on Fragile X Syndrome. It also includes an overview of trial numbers and their average enrollment in top countries conducted across the globe. Moreover, it offers coverage of disease clinical trials by region, country (G7 & E7), phase, trial status, end points...
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Product Insights
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Adrenoleukodystrophy disease is caused by a defect in the gene that tells the body to create lipoproteins. It mainly affects the nervous system and the adrenal glands, which are small glands located on top of each kidney. The Adrenoleukodystrophy pipeline market research report provides comprehensive information on the therapeutics under development for Adrenoleukodystrophy, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. The report also covers the descriptive pharmacological...
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Spinocerebellar Ataxia (SCA) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Spinocerebellar ataxias (SCAs) are a group of inherited conditions that affect the brain and spinal cord causing progressive difficulty with coordination. Its symptoms include poor coordination, unsteady walking, change in speech, and difficulty swallowing. The Spinocerebellar Ataxia pipeline market research report provides comprehensive information on the therapeutics under development for Spinocerebellar Ataxia, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. The report also covers the descriptive pharmacological action...
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Polycystic Kidney Disease Clinical Trial Analysis by Trial Phase, Trial Status, Trial Counts, End Points, Status, Sponsor Type, and Top Countries, 2022 Update
The Polycystic Kidney Disease clinical trial market research report provides an overview of the Polycystic Kidney Disease clinical trials scenario. The report provides top-line data relating to the clinical trials on Polycystic Kidney Disease. The Polycystic Kidney Disease, 2022 report includes an overview of trial numbers and their average enrollment in top countries conducted globally. The clinical trials report also includes coverage of disease clinical trials by region and top countries, trial phase, trial status, end points status and sponsor...
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Product Insights
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Drugs In Development, 2022, provides an overview of the Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders) pipeline landscape. Niemann-Pick C disease is one of a group of lysosomal storage diseases that affect metabolism and that are caused by genetic mutations. It involves the accumulation of sphingolipids in cells throughout the body, particularly reticuloendothelial cells (the mononuclear phagocyte system). Symptoms include...
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Product Insights
Neurofibromatoses Type II Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Neurofibromatoses Type II – Drugs In Development, 2022, provides an overview of the Neurofibromatoses Type II (Genetic Disorders) pipeline landscape. Neurofibromatosis type 2 is a disorder characterized by the growth of noncancerous tumors in the nervous system. These growths develop along the nerve that carries information from the inner ear to the brain (the auditory nerve). Tumors that occur on other nerves are also commonly found with this condition. The...
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Product Insights
Polycystic Kidney Disease Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Polycystic Kidney Disease – Drugs In Development, 2022, provides an overview of the Polycystic Kidney Disease (Genetic Disorders) pipeline landscape. Polycystic kidney disease (PKD) is a disorder in which clusters of cysts develop primarily within kidneys. Polycystic kidney disease symptoms may include high blood pressure, back or side pain, headache, blood in urine, frequent urination and kidney failure. The predisposing factors include age and family history. Treatment includes antihypertensive drugs...
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Sector Analysis
Niemann-Pick Type C Market Size and Trend Report including Epidemiology and Pipeline Analysis, Competitor Assessment, Unmet Needs, Clinical Trial Strategies and Forecast, 2021-2031
The Niemann-Pick Type C (NPC) market size was $128.35 million in 2021 and is forecast to achieve a CAGR of more than 3% during the forecast period. Major symptoms of the disease are neurological and developmental disorders, as well as hepatosplenomegaly, jaundice, cholestasis, bleeding disorders, and eventually dysphagia and even respiratory failure in adults. Early diagnosis remains a critically unmet need, with many patients receiving a diagnosis late after the onset of neurological disease and other musculoskeletal disabilities.
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Product Insights
Fragile X Syndrome Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Fragile X syndrome is caused by a change in a gene called FMR1. It occurs both in males and females. The symptoms of Fragile X Syndrome include delay in crawling, walking, or twisting, hyperactive or impulsive behavior, speech or language delay, and intellectual disability. The Fragile X Syndrome pipeline market research report provides comprehensive information on the therapeutics under development for Fragile X Syndrome, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration...
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Down Syndrome Clinical Trial Analysis by Trial Phase, Trial Status, Trial Counts, End Points, Status, Sponsor Type, and Top Countries, 2022 Update
Down syndrome clinical trials market research report provides an overview of the Down Syndrome dystrophy clinical trials scenario. This report provides top-line data relating to the clinical trials on Down Syndrome dystrophy. The report includes an overview of trial numbers and their average enrolment in top countries conducted across the globe. The report offers coverage of disease clinical trials by region, country (G7 & E7), phase, trial status, end points status, and sponsor type. The report also provides prominent drugs...
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Sector Analysis
Aicardi-Goutieres Syndrome Marketed and Pipeline Drugs Assessment, Clinical Trials and Competitive Landscape
Aicardi-Goutieres syndrome pipeline market report combines data from the Pharma Intelligence Center with in-house analyst expertise to provide a competitive assessment of the disease marketplace. Aicardi-Goutieres syndrome (AGS) is an inherited disease that mainly affects the brain, immune system, and the skin. Loss of white matter in the brain (leukodystrophy) and abnormal deposits of calcium (calcification) in the brain lead to early-onset severe brain dysfunction (encephalopathy) that usually results in severe intellectual and physical disability.
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Sector Analysis
Alport Syndrome Marketed and Pipeline Drugs Assessment, Clinical Trials and Competitive Landscape
The Alport syndrome pipeline drugs market research report includes an assessment of the disease epidemiology, leading marketed products, late-to-mid-stage pipeline products with Phase Transition Success Rate (PTSR), and Likelihood of Approval (LoA) for Alport syndrome. Â The report also analyzes the clinical and commercial landscapes of Alport syndrome, as well as an assessment of deals, future market catalysts, key market players, and the future competitive environment. Alport syndrome (AS) is a genetically and phenotypically heterogeneous, progressive hereditary renal disease that is...
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Product Insights
Netherton Syndrome (Trichorrhexis Invaginata, Bamboo Hair) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players
Netherton Syndrome (Trichorrhexis Invaginata, Bamboo Hair) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players provides an overview of the Netherton Syndrome (Trichorrhexis Invaginata, Bamboo Hair) pipeline landscape. The report provides comprehensive information on the therapeutics under development for Netherton Syndrome (Trichorrhexis Invaginata, Bamboo Hair), complete with analysis by Stage of Development, Drug Target, Mechanism of Action (MoA), Route of Administration (RoA) and Molecule Type. The report also covers the descriptive Pharmacological Action of the therapeutics,...
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Product Insights
Charcot-Marie-Tooth Disease Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players
Charcot-Marie-Tooth Disease Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players provides an overview of the Charcot-Marie-Tooth Disease pipeline landscape. The report provides comprehensive information on the therapeutics under development for Charcot-Marie-Tooth Disease, complete with analysis by Stage of Development, Drug Target, Mechanism of Action (MoA), Route of Administration (RoA) and Molecule Type. The report also covers the descriptive Pharmacological Action of the therapeutics, its complete research and development history and latest news and press releases....
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