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


 Wolfram syndrome, also referred to as DIDMOAD, is a hereditary condition commonly characterized by childhood-onset insulin-dependent diabetes mellitus and progressive optic atrophy. It is caused by mutations in two genes: WFS1 (4p16.1) and CISD2 (4q24). WFS1 produces Wolframin, a protein in the endoplasmic reticulum (ER) that maintains calcium levels and responds to unfolded proteins. Mutations in WFS1 are primarily responsible for most Wolfram syndrome symptoms, defining WS1. CISD2, on the other hand, encodes the ERIS protein, which moves dynamically between the ER and the outer membrane of mitochondria, regulating glucose homeostasis, insulin sensitivity, calcium balance, and autophagy. Mutations in CISD2 lead to WS2. Common symptoms of Wolfram syndrome encompass diabetes mellitus, optic nerve deterioration, central diabetes insipidus, sensorineural hearing loss, urinary tract issues, and progressive neurological challenges. Diabetes mellitus is the initial sign, often diagnosed around the age of 6, followed by optic nerve damage characterized by diminished color and peripheral vision by around age 11. Central diabetes insipidus affects approximately 70% of Wolfram patients. Sensorineural hearing loss occurs in about 65% of cases, varying from congenital deafness to gradual hearing loss starting in adolescence. Neurological issues, particularly ataxia, commonly arise in early adulthood in more than 60% of individuals with Wolfram syndrome. Presently, there are no treatments capable of halting or reversing the progression of Wolfram syndrome. However, diligent clinical monitoring and supportive care can alleviate symptoms and enhance patients' quality of life. Supportive management includes regular screening for diabetes mellitus, vision impairment, urinary function, kidney health, and insulin injections, along with dietary control for diabetes. Additionally, management involves addressing central diabetes insipidus, respiratory issues, urinary problems, and psychological well-being through prophylactic measures, antibiotic therapy for urinary infections, and periodic evaluation for depression and other psychiatric symptoms.

·       Wolfram syndrome type 1 is estimated to affect 1 in 500,000 people globally, with approximately 200 reported cases in scientific literature. Wolfram syndrome type 2 is rare, with only a few Jordanian families identified with this condition.

 

Thelansis’s “Wolfram Syndrome 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 Wolfram Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

 

KOLs insights of Wolfram Syndrome 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.

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

Tags: Wolfram Syndrome, Wolfram Syndrome market outlook, Wolfram Syndrome competitive landscape, Wolfram Syndrome market forecast, Thelansis, Primary market research, KOL insights, Competitive Intelligence (CI)

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