Inherited Metabolic Disorders
Discover unique market-leading data and insights into the Inherited Metabolic Disorders market. From the latest Inherited Metabolic Disorders industry reports and analysis to comprehensive market growth forecasts, trends and research.
Browse our full list of Inherited Metabolic Disordersmarket reports here.
- Currency Conversion is for Indicative purpose only. All orders are processed in US Dollars only.
- USD - US Dollar
- AUD — Australian Dollar
- BRL — Brazilian Real
- CNY — Yuan Renminbi
- EUR — Euro
- GBP — Pound Sterling
- INR — Indian Rupee
- JPY — Japanese Yen
- ZAR — South African Rand
- The Price Conversion is only for the Indicative purpose.
- USD — US Dollar
- AUD — Australian Dollar
- BRL — Brazilian Real
- CNY — Yuan Renminbi
- EUR — Euro
- GBP — Pound Sterling
- INR — Indian Rupee
- JPY — Japanese Yen
- ZAR — South African Rand
-
Product Insights
NewProduct Insights NewGaucher Disease Clinical Trial Analysis by Phase, Trial Status, End Point, Sponsor Type and Region, 2024 Update Gaucher Disease Clinical Trial Analysis by Phase, Trial Status, End Point, Sponsor Type and Region, 2024 Update
$2,500 | May 2024 GlobalData's clinical trial report, “Gaucher Disease - Global Clinical Trials Review, 2024" provides an overview of Gaucher Disease Clinical trials scenario. This report provides top line data relating to the clinical trials on Gaucher Disease. 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 trials by region, country (G7 & E7), phase, trial status, end points status and sponsor type. Report also provides prominent...
-
Product Insights
Product Insights Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Clinical Trial Analysis by Phase, Trial Status, End Point, Sponsor Type and Region, 2024 Update Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Clinical Trial Analysis by Phase, Trial Status, End Point, Sponsor Type and Region, 2024 Update
$2,500 | March 2024 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) Clinical Trial Report Overview A total of 97 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) clinical trials were conducted as of March 2024. The Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) clinical trial report provides a comprehensive understanding of the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) clinical trial scenario across regions, and countries (G7 & E7), along with insights into the various phases, trial status, and end points status. The report also includes information about the...
-
Product Insights
Product Insights Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Clinical Trial Analysis by Phase, Trial Status, End Point, Sponsor Type and Region, 2024 Update Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Clinical Trial Analysis by Phase, Trial Status, End Point, Sponsor Type and Region, 2024 Update
$2,500 | February 2024 Mucopolysaccharidosis II (MPS II) Clinical Trial Report Overview A total of 84 Hunter syndrome clinical trials were conducted as of February 2024. The MPS II clinical trial report provides a comprehensive understanding of the Mucopolysaccharidosis II clinical trial scenario across regions, and countries (G7 & E7), along with insights into the various phases, trial status, and end points status. The report also includes information about the sponsor types and the prominent sponsors associated with the trials. Key Regions ·       North...
-
Product Insights
NewProduct Insights NewAcid Sphingomyelinase Deficiency (Niemann-Pick Disease) – Drugs In Development, 2024 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) – Drugs In Development, 2024
$2,000 | June 2024 Empower your strategies with our Niemann-Pick Disease – Drugs In Development, 2024 report and make more profitable business decisions. Acid sphingomyelinase deficiency (ASMD) is a rare genetic disorder that affects the metabolism of a lipid called sphingomyelin. ASMD is caused by mutations in the SMPD1 gene, which encodes the enzyme acid sphingomyelinase (ASM). ASM is responsible for breaking down sphingomyelin in the lysosomes of cells. When ASM is deficient, sphingomyelin accumulates in various tissues and organs, causing damage and dysfunction. ASMD...
-
Product Insights
NewProduct Insights NewMucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Drugs In Development, 2024 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Drugs In Development, 2024
$2,000 | June 2024 Empower your strategies with our Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Drugs In Development, 2024 report and make more profitable business decisions. Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is an inherited metabolic disease caused by an absence or malfunctioning of certain enzymes needed to break down molecules called glycosaminoglycans (GAGs). Symptoms include seizures, hyperactivity, liver and spleen enlargement, severe diarrhea or constipation, and enlargement of tonsils and adenoids. Treatment includes enzyme replacement therapy (ERT). The...
-
Product Insights
NewProduct Insights NewMucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Drugs In Development, 2024 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Drugs In Development, 2024
$2,000 | June 2024 Empower your strategies with our Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Drugs In Development, 2024 report and make more profitable business decisions. Mucopolysaccharidosis type I (MPS I) is an inherited lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness, and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy, and...
-
Product Insights
NewProduct Insights NewFatty Acid Biosynthesis Disorders – Drugs In Development, 2024 Fatty Acid Biosynthesis Disorders – Drugs In Development, 2024
$2,000 | June 2024 Empower your strategies with our Fatty Acid Biosynthesis Disorders – Drugs In Development, 2024 report and make more profitable business decisions. Fatty acid biosynthesis disorders (FABDs) are a group of inherited metabolic disorders that affect the ability of the body to use fatty acids as a source of energy. Fatty acids are long chains of carbon and hydrogen atoms that are stored in fat cells and can be broken down to produce energy when glucose (sugar) is not available. However, people...
-
Product Insights
NewProduct Insights NewAcid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Drugs In Development, 2024 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Drugs In Development, 2024
$2,000 | June 2024 Empower your strategies with our Niemann-Pick Disease Type C – Drugs In Development, 2024 report and make more profitable business decisions. Niemann-Pick 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 enlarged liver, brain damage, difficulty walking and swallowing, increased sensitivity to touch, difficulty speaking, loss of muscle tone, and...
-
Product Insights
NewProduct Insights NewLipid Storage Disorders (Lipidoses) – Drugs In Development, 2024 Lipid Storage Disorders (Lipidoses) – Drugs In Development, 2024
$2,000 | June 2024 Empower your strategies with our Lipid Storage Disorders (Lipidoses) – Drugs In Development, 2024 report and make more profitable business decisions. Lipid storage disorders, or lipidoses, encompass inherited metabolic conditions where fats accumulate due to enzyme deficiencies, impacting various organs. Gaucher, Niemann-Pick, Tay-Sachs, Fabry, and Krabbe diseases are examples. Symptoms, varying by disorder, may include neurological problems, organ enlargement, and skeletal abnormalities. Treatments focus on symptom management, employing enzyme replacement, substrate reduction therapies, and supportive care. Managing these complex conditions requires...
-
Product Insights
NewProduct Insights NewGaucher Disease – Drugs In Development, 2024 Gaucher Disease – Drugs In Development, 2024
$2,000 | June 2024 Empower your strategies with our Gaucher Disease – Drugs In Development, 2024 report and make more profitable business decisions. Gaucher disease is a rare genetic disorder that occurs due to the lack of an enzyme called glucocerebrosidase. Symptoms include cognitive impairment, enlarged spleen, severe swelling, lung disease, fatigue, and seizures. Treatment includes enzyme replacement therapy. The Gaucher Disease drugs in development market research report provide comprehensive information on the therapeutics under development for Gaucher Disease, complete with analysis by stage of...
![](https://d2ciohgjvuch9f.cloudfront.net/store/wp-content/themes/html5blank/assets/img/marketing/report-banner-newsletter.webp)
Sign up to receive regular alerts for our latest analysis and reports. No matter your industry focus, you can keep your finger on the pulse with our timely updates.