Maple Syrup Urine Disease (MSUD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032


 Maple syrup urine disease (MSUD) is a rare inborn metabolic error characterized by reduced branched-chain ketoacid dehydrogenase enzyme activity. This deficiency can lead to severe irreversible neurocognitive deficits, underlining the importance of prompt diagnosis and treatment to mitigate the severity of complications. The branched-chain ketoacid dehydrogenase (BCKAD) complex is comprised of four subunits. These include the E1 subunit (BCKAD decarboxylase), the E2 subunit (dihydrolipoyl acyltransferase), and the E3 subunit (dihydrolipoamide dehydrogenase). Additionally, regulatory subunits known as BCKAD kinase and BCKAD phosphatase are associated with the core of the E2 subunit. The E1 subunit consists of E1alpha and two E1beta subunits. The root cause of the metabolic disorder lies in the decreased activity of the branched-chain alpha-ketoacid dehydrogenase complex located within the mitochondria. This disorder is inherited in an autosomal recessive manner. Maple syrup urine disease manifests in five primary clinical phenotypes. There exist five clinical subtypes of MSUD: classic, Intermediate, Intermittent, Thiamine responsive, and E3 deficient. These subtypes are differentiated based on their clinical presentation, age of onset, and the remaining BCKAD enzyme activity. It is crucial to distinguish from other conditions that can present as neonatal encephalopathy. These include Hypoglycemia, Status epilepticus, Meningitis, Encephalitis, Kernicterus, Birth asphyxia, Urea cycle defects, and Organic acidopathies such as propionic acidemia and methylmalonic acidemia, HMG-CoA lyase deficiency, Beta-ketothiolase deficiency, and Non-ketotic hyperglycinemia. The treatment approach involves restricting dietary leucine, utilizing BCAA-free medical foods, supplementing judiciously with isoleucine and valine, and closely monitoring clinical and biochemical indicators. Catabolic illnesses can be managed in outpatient settings through a "sick-day" formula, frequent amino acid monitoring, stress management, and targeted nutritional support. The prognosis is more favorable for patients who commence therapy before or immediately after symptom onset. Plasma leucine concentrations influence neurocognitive outcomes. Notably, individuals with classic MSUD have demonstrated better cognitive performance in school-aged settings. Many adult MSUD patients can lead independent lives and integrate well into society, although psychological and psychiatric care is still required for a substantial portion. Late-onset MSUD cases might experience mild developmental delays depending on residual BCKAD activity. A delayed diagnosis of classic MSUD beyond 7 to 14 days can result in irreversible learning impairment and cerebral palsy.

·       The calculated incidence of maple syrup urine disease in the United States is approximately 1 in 220,219 individuals.

Thelansis’s “Maple Syrup Urine Disease (MSUD) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032" 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 Maple Syrup Urine Disease (MSUD) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

KOLs insights of Maple Syrup Urine Disease (MSUD) 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.

Maple Syrup Urine Disease (MSUD) 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: Maple Syrup Urine Disease (MSUD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

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

Herpes Simplex Virus (HSV) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Image
 Herpes simplex virus (HSV) causes an infection that affects the external genitalia, anal region, mucosal surfaces, and skin in other body parts. Herpes simplex virus is a ubiquitous, host-adapted pathogen that causes various disease states. There are two types of HSV with two distinct DNA viruses, type 1 (HSV-1) and type 2 (HSV-2). Both the types are closely related but differ epidemiologically. HSV-1 is traditionally associated with orofacial disease by affecting the mouth and lips with fever blisters and cold sores, whereas HSV-2 is associated traditionally with genital disease infecting genitals and rectum area with sores and is classified as STD (sexually transmitted disease). As HSV-1 is associated with genital infections more often than HSV-2, lesion location is not necessarily of viral type. HSV can spread through direct contact. Almost 80% of Herpes simplex infections are asymptomatic, while symptomatic infections can be characterized by significant morbidity and recurrenc...
Read more

HR+/HER2- Breast Cancer – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Image
Thelansis’s “HR+/HER2- Breast Cancer 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 HR+/HER2- Breast Cancer treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China). KOLs insights of HR+/HER2- Breast Cancer 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. HR+/HER2- Breast Cancer 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,...
Read more