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Wolfram Syndrome (WS) | Primary Research (KOL’s Insight) | Market Intelligence | Epidemiology & Market Forecast-2030

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Novel therapies designed for this ultra-rare disorder, Wolfram Syndrome (WS) may have broader implications for more common medical conditions related to ER stress and dysfunction.  The tools and therapeutic efforts targeting Wolfram syndrome has benefitted in identifying novel treatment modalities for more prevalent disorders such as diabetes mellitus and neurodegenerative disease.
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Description

Wolfram syndrome is an autosomal recessive genetic disorder, characterized by diabetes insipidus (DI), childhood-onset diabetes mellitus (DM), a gradual loss of vision caused by optic atrophy (OA), deafness (D; hence the acronym: DIDMOAD), and neurological signs. Other symptoms include bladder, bowel and temperature regulation dysfunctions, endocrinological, psychiatric, and neurological abnormalities

Wolfram (DIDMOAD) syndrome Epidemiology

Globally, the estimated prevalence of Wolfram Syndrome (WS) type 1 is 1 in 500,000 people. According to the N. B. Toppings et. al. 2018, estimated prevalence of WS falls between 1 in 770,000 in the United Kingdom and 1 in 100,000 in North America. About 60 percent of people with Wolfram Syndrome (WS) develop a neurological or psychiatric disorder, most commonly problems with balance and coordination (ataxia), typically beginning in early adulthood.

The total prevalent cases in the G7 countries are anticipated to rise to ~XXX by 2030 at a CAGR of 0.7% for the study period (2017–2030). The diagnosed prevalent and drug-treated population of Wolfram Syndrome in the G7 countries was XXX cases and XXX cases respectively.

WS is associated with multiple clinical associated conditions i.e. Diabetes mellitus <16 yrs, Optic atrophy <16 yrs, Diabetes insipidus, Diabetes mellitus >16yrs, Optic atrophy >16 yrs, Sensorineural deafness, Neurological signs (ataxia, epilepsy, cognitive impairment) and Renal tract abnormalities (structural or functional). The large number (<80%) of WS cases were found to be associated with Diabetes mellitus <16 yrs, and Optic atrophy <16 yrs.

Wolfram-syndrome-Cases-by-Clinical-Manifestation- Mellalta Meets

Wolfram (DIDMOAD) syndrome Market Forecast

The Wolfram Syndrome (WS) therapy market is projected to experience moderate growth during our study period, rising to substantial $ million market size in 2030, representing an annual growth of 53%. The primary drivers of this growth will be the adoption of technological advancements, standard diagnostic tools for detection of the rare diseases, rising awareness, and accessibility to healthcare infrastructure along with an increase in the number of incident Wolfram Syndrome (WS) cases.

Currently there is no cure for Wolfram Syndrome (WS), and current treatment aims to manage the underlying cause and prognosis of Wolfram Syndrome (WS). However, the future landscape for the treatment of WS looks brighter because even though the disease is an ultra-rare genetic disorder, its constituent medical components (e.g. diabetes mellitus, deafness, and retinal degeneration) and underlying ER pathophysiology are individually present in many more common diseases. Consequently, novel treatments designed for WS may have broader implications for more common medical conditions related to ER stress and dysfunction. Thus, by leveraging the tools and therapeutic efforts targeting Wolfram syndrome, researchers may very well identify novel treatment modalities for more prevalent disorders such as diabetes mellitus and neurodegenerative diseases. ​

One of the novel approaches to provide a cure to Wolframs syndrome could be via gene therapy. Using adeno-associated viral (AAV) systems, wild-type WFS1 could be transferred into the retinal ganglion cells of patients with Wolfram syndrome to supplement the production of correct protein in the human body. AMX0035  is currently in development by Amylyx Pharma to target the ER stress, and preclinical data suggest that it may be a promising approach to halt the irreversible progression of optic nerve atrophy in patients with Wolfram syndrome. AMX0035 targets endoplasmic reticulum stress (a molecular mechanism of Wolfram) and mitochondria dysfunction.

Wolfram Syndrome Market Overview- Country wise Analysis 2030 Mellalta Meets

Report Highlights

  • Wolfram syndrome Current Market Trends
  • Wolfram syndrome Current & Forecasted Cases across the G7 Countries
  • Wolfram syndrome Market Opportunities And Sales Potential for Agents
  • Wolfram syndrome Patient-based Market Forecast to 2030
  • Wolfram syndrome Untapped Business Opportunities
  • Wolfram syndrome Product Positioning Vis-a-vis Competitors’ Products
  • Wolfram syndrome KOLs Insight

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Table of Contents

  • Executive Summary
    • Key Findings
    • Key Challenges & Opportunities
  • Wolfram Syndrome (WS) Disease Background
    • Wolfram Syndrome (WS) Disease Definition
    • Symptoms & Causes
    • Wolfram Syndrome: Common Clinical Presentations
    • Wolfram syndrome Differential Diagnosis
  • Epidemiology and Patient Populations
    • Key Findings
    • Methods and data Sources
      • Country Specific Prevalent Populations (US, Germany, France, Italy, Spain, UK, and Japan)
      • Country Specific Clinical Manifestations Associated Wolfram Syndrome (WS) Cases
      • Country Specific Diagnosed and Treated Cases of Wolfram Syndrome (WS)
    • Number of Diagnosed and Drug-Treated Cases of Wolfram Syndrome (WS) in the Major Pharmaceutical Markets, 2017-2030
    • Key Sources for Wolfram Syndrome (WS) Epidemiology and Model Parameters
      • United States
        • United States Prevalent Populations of Wolfram Syndrome (WS)
        • United States Clinical Manifestations Associated Cases of Wolfram Syndrome (WS)
        • United States Diagnosed and Treated Cases of Wolfram Syndrome (WS)
      • Germany
        • Germany Prevalent Populations of Wolfram Syndrome (WS)
        • Germany Clinical Manifestations Associated Cases of Wolfram Syndrome (WS)
        • Germany Diagnosed and Treated Cases of Wolfram Syndrome (WS)
      • France
        • France Prevalent Populations of Wolfram Syndrome (WS)
        • France Clinical Manifestations Associated Cases of Wolfram Syndrome (WS)
        • France Diagnosed and Treated Cases of Wolfram Syndrome (WS)
      • Italy
        • Italy Prevalent Populations of Wolfram Syndrome (WS)
        • Clinical Manifestations Associated Cases of Wolfram Syndrome (WS)
        • Italy Diagnosed and Treated Cases of Wolfram Syndrome (WS)
      • Spain
        • Spain Prevalent Populations of Wolfram Syndrome (WS)
        • Spain Clinical Manifestations Associated Cases of Wolfram Syndrome (WS)
        • Spain Diagnosed and Treated Cases of Wolfram Syndrome (WS)
      • United Kingdom
        • United Kingdom Prevalent Populations of Wolfram Syndrome (WS)
        • United Kingdom Clinical Manifestations Associated Cases of Wolfram Syndrome (WS)
        • United Kingdom Diagnosed and Treated Cases of Wolfram Syndrome (WS)
      • Japan
        • Japan Prevalent Populations of Wolfram Syndrome (WS)
        • Japan Clinical Manifestations Associated Cases of Wolfram Syndrome (WS)
  • Japan Diagnosed and Treated Cases of Wolfram Syndrome (WS)
  • Current Therapies and Medical Practice
    • Treatments & Medical Practices
    • Treatment patterns in Wolfram Syndrome
  • Cost of illness (COI) associated with Wolfram syndrome
  • Survey highlighting Psychiatric Diagnoses and Treatment in Wolfram Syndrome
  • Unmet Needs
  • Emerging Therapies
    • Pipeline Overview
    • Therapeutic Developments Pipeline for Wolfram Syndrome (WS)
      • Product Analysis
        • Depakine (Genethon)
          • Product Profile
          • Clinical Development
        • AMX0035 (Amylyx)
          • Product Profile
        • AMRS001 (Amarantus BioScience))
          • Product Development
  • Future Treatment Paradigm
    • Wolfram Syndrome (WS) Competitor Landscape and Approvals Anticipated
    • Future Treatment Algorithms and Competitor Positioning
    • Key Data Summary for Emerging Treatment
  • Annual Cost of Current & Emerging Therapies
  • Market Outlook
    • Key Findings
    • Country Specific Market Forecast to 2030
      • Sales of Drugs to Treat Wolfram Syndrome (WS) in the Major Pharmaceutical Markets, 2017-2030
      • Patient Share of Wolfram Syndrome (WS) Therapies and by Drug
  • Market Forecast by Country
    • United States
      • United States Market for Wolfram Syndrome (WS) 2020-2030 (USD Million)
      • United States Market for Wolfram Syndrome (WS) by Therapies 2020-2030 (USD Million)
    • Germany
      • Germany Market for Wolfram Syndrome (WS) 2020-2030 (USD Million)
      • Germany Market for Wolfram Syndrome (WS) by Therapies 2020-2030 (USD Million)
    • France
      • France Market for Wolfram Syndrome (WS) 2020-2030 (USD Million)
      • France Market for Wolfram Syndrome (WS) by Therapies 2020-2030 (USD Million)
    • Italy
      • Italy Market for Wolfram Syndrome (WS) 2020-2030 (USD Million)
      • Italy Market for Wolfram Syndrome (WS) by Therapies 2020-2030 (USD Million)
    • Spain
      • Spain Market for Wolfram Syndrome (WS) 2020-2030 (USD Million)
      • Spain Market for Wolfram Syndrome (WS) by Therapies 2020-2030 (USD Million)
    • United Kingdom
      • United Kingdom Market for Wolfram Syndrome (WS) 2020-2030 (USD Million)
      • United Kingdom Market for Wolfram Syndrome (WS) by Therapies 2020-2030 (USD Million)
    • Japan
      • Japan Market for Wolfram Syndrome (WS) 2020-2030 (USD Million)
      • Japan Market for Wolfram Syndrome (WS) by Therapies 2020-2030 (USD Million)
  • Market Drivers and Constraints
    • What Factors Are Driving the Market for Wolfram Syndrome (WS)?
    • What Factors Are Constraining the Market for Wolfram Syndrome (WS)?
    • Appendix
      • Report Methodology
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