Wolfram Syndrome – Market outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2020 To 2030

Wolfram syndrome, also known by the acronym DIDMOAD, is an inherited condition typically associated with childhood-onset insulin-dependent diabetes mellitus and progressive optic atrophy. In addition, many people with Wolfram syndrome also develop diabetes insipidus and sensorineural hearing loss.

Etiology-

The two types of Wolfram syndrome (type 1 and type 2) are primarily differentiated by their genetic cause.  Variations (mutations) in the WFS1 gene are responsible for about 90% of Wolfram syndrome type 1 cases. This gene encodes wolframin, an essential protein for the endoplasmic reticulum's proper functioning (the part of a cell involved in protein production, processing, and transport). Wolframin helps regulate the amount of calcium in cells, which is essential for many different cellular functions.

A specific mutation in the CISD2 gene causes Wolfram syndrome type 2. Although the exact function of this gene is not known, scientists suspect that it plays a vital role in the mitochondria (the part of the cell where energy is produced)—mutations in CISD2 lead to the loss of mitochondria which decreases the amount of energy available to cells.

Epidemiology-

The estimated prevalence of Wolfram syndrome type 1 is 1 in 500,000 people worldwide. Approximately 200 cases have been described in the scientific literature. Only a few families from Jordan have been found to have Wolfram syndrome type 2.

The competitive landscape of Wolfram Syndrome includes country-specific approved and pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are tracked and supplemented with analyst commentary.

KOLs insights of Wolfram Syndrome across 8 MM market from the center 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.

Read more: Wolfram Syndrome – Market outlook, Epidemiology, Market Forecast, and Competitive Landscape Report – 2020 To 2030

S. No    Asset               Company                                 Stage

1          AMX0035          Amylyx Pharmaceuticals            Phase 1

 

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