Hurler Syndrome (Mucopolysaccharidosis Type I) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Mucopolysaccharidosis type I (MPS I), or Hurler Syndrome, is a genetic condition that impacts various body parts. It is caused by alterations in the IDUA gene, resulting in a deficiency or complete absence of the IDUA enzyme. In the absence of this enzyme, large sugar molecules, known as glycosaminoglycans (GAGs), accumulate within cellular structures called lysosomes. This accumulation causes lysosomes to increase, leading to the enlargement of multiple organs and tissues in the body, which is responsible for the observed medical issues associated with the condition. Typically, the clinical onset of MPS I occurs between three and ten. The severity and progression of the disease can vary widely, ranging from severe, life-threatening complications that may lead to death in the second or third decade of life to a relatively longer but compromised lifespan characterized by significant disability resulting from progressive joint problems and cardiorespiratory issues. One available treatment option is hematopoietic stem cell transplantation (HSCT). This procedure can enhance cognitive function, increase survival rates, promote growth, reduce facial coarseness and hepatosplenomegaly (enlargement of the liver and spleen), improve hearing, prevent hydrocephalus (excess fluid in the brain), and alter the natural course of cardiac and respiratory symptoms.

·       The incidence of Mucopolysaccharidosis type I (MPS I) ranges from 1 to 1.5 cases per 100,000 live births for the severe type and an incidence of about 1 in the 500,000 live births for the attenuated type.

 

Thelansis’s “Hurler Syndrome (Mucopolysaccharidosis Type I) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Hurler Syndrome (Mucopolysaccharidosis Type I) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

 

KOLs insights of Hurler Syndrome (Mucopolysaccharidosis Type I) across 8 MM market from the centre of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

Hurler Syndrome (Mucopolysaccharidosis Type I) Market Forecast Patient Based Forecast Model (MS. Excel Based Automated Dashboard), which Data Inputs with sourcing, Market Event, and Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.

Thelansis Competitive Intelligence (CI) practice has been established based on a deep understanding of the pharma/biotech business environment to provide an optimized support system to all levels of the decision-making process. It enables business leaders in forward-thinking and proactive decision-making. Thelansis supports scientific and commercial teams in seamless CI support by creating an AI/ ML-based technology-driven platform that manages the data flow from primary and secondary sources.

Read more: Hurler Syndrome (Mucopolysaccharidosis Type I) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Tags: Hurler Syndrome (Mucopolysaccharidosis Type I), Hurler Syndrome (Mucopolysaccharidosis Type I) market outlook, Hurler Syndrome (Mucopolysaccharidosis Type I) competitive landscape, Hurler Syndrome (Mucopolysaccharidosis Type I) market forecast, Thelansis, Primary market research, KOL insights, Competitive Intelligence (CI)

Comments

Post a Comment

Popular posts from this blog

Acne Vulgaris – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

Image
  Acne vulgaris, a prevalent pilosebaceous unit inflammatory skin disorder, follows a persistent course. Typically presenting as papules, pustules, or nodules, it primarily occurs on the face but can also impact the upper arms, trunk, and back. The development of acne results from the sebaceous glands' heightened sensitivity to normal androgen levels, aggravated by Cutibacterium acnes (formerly known as Propionibacterium acnes), a bacterial species that triggers inflammation. The pathogenesis of acne vulgaris involves a complex interplay of factors such as the activation of sebaceous glands by androgens, imbalances in the pilosebaceous follicle microbiome, and cellular immune responses. Genetic predisposition and dietary influences also contribute to the condition's onset and progression. The initial stage is the micro-comedo, a small plug primarily composed of corneocytes in the lower follicular infundibulum. Microcomedones evolve into various acne lesions, including closed co
Read more

Metabolic Syndrome – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Image
  Metabolic syndrome refers to a clustering of multiple disorders that collectively increase an individual's susceptibility to atherosclerotic cardiovascular disease, insulin resistance, diabetes mellitus, as well as vascular and neurological complications such as cerebrovascular accidents. The underlying causes of metabolic syndrome typically include excess weight, obesity, a sedentary lifestyle, and genetic predisposition. This syndrome exerts detrimental effects on various body systems. Insulin resistance, a key component of metabolic syndrome, leads to microvascular damage, increasing the risk of endothelial dysfunction, vascular resistance, hypertension, and inflammation of vessel walls. Bariatric surgery is recognized as the most effective standalone therapy for metabolic syndrome. However, patients undergoing this procedure require long-term follow-up to mitigate potential surgical, nutritional, and psychiatric complications. Thelansis’s “Metabolic Syndrome Market Outl
Read more

Type 1 Diabetes Mellitus (T1D) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Image
  Type 1 Diabetes Mellitus (T1D) is characterized as an autoimmune disorder resulting in the autoimmune destruction of insulin-producing pancreatic beta cells. This condition necessitates life-long insulin replacement, administered through multiple daily insulin injections, insulin pump therapy, or an automated insulin delivery system. The absence of insulin can lead to the development of Diabetic Ketoacidosis (DKA), a life-threatening condition. The most common symptoms associated with this disease are polyuria and polydipsia. Effective management of T1D involves: Multiple daily insulin injections Insulin pump therapy An automated insulin delivery system in conjunction with glucose monitoring Continuous Glucose Monitoring (CGM) is the preferred method. However, all individuals with T1D should be capable of performing Blood Glucose Monitoring (BGM) via capillary blood if CGM is not available. Acute complications of diabetes primarily include hypoglycemia and severe
Read more