Sorbitol Dehydrogenase (SORD) Deficiency – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

Sorbitol Dehydrogenase (SORD) Deficiency Market Outlook

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

Sorbitol Dehydrogenase (SORD) Deficiency Overview

Sorbitol dehydrogenase deficiency (SORD Deficiency) is a progressive hereditary neuropathy that significantly impacts peripheral nerves and motor neurons, leading to considerable disability, sensory loss, and reduced mobility. Consequently, intracellular sorbitol accumulates, and this molecular alteration has been identified as the underlying cause of the disease in a specific subgroup of patients previously diagnosed with Charcot-Marie-Tooth disease (CMT2) or distal hereditary motor neuropathy (dHMN). A subtype of Charcot-Marie-Tooth disease (CMT) is caused by a mutated SORD gene, which encodes for sorbitol dehydrogenase. The mutated gene results in elevated levels of sorbitol, leading to axonal damage in the peripheral nerves. Individuals with SORD Deficiency experience a progressive form of neuropathy, typically commencing in late adolescence. This condition can substantially impede the individual's quality of life, presenting symptoms such as limb weakness, foot deformities, tremors, sensory impairment, and difficulties with walking. Onset of SORD Deficiency symptoms typically occurs around the age of 17, although it can arise anytime between 9 and 25 years of age. Clinical diagnosis of SORD Deficiency relies on the presence of a slowly progressing neuropathy, often accompanied by foot deformities. In addition to clinical assessment of signs, symptoms, and neurophysiological evaluations, SORD Deficiency can also be confirmed through genetic testing and motor nerve conduction velocity (MNCV) testing. Currently, there are no approved pharmacological treatments for SORD Deficiency. Management of symptoms involves a multidisciplinary approach, including rehabilitative measures, physical and occupational therapy, and surgical interventions in cases of severe impairment.

• In the United States, it is estimated that there are around 3,300 individuals who carry mutations in the SORD gene that contribute to the development of the disorder.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2022-2032

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets 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

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- 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

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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