X-linked Adrenoleukodystrophy (X-ALD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

X-linked Adrenoleukodystrophy (X-ALD) Market Outlook

Thelansis’s “X-linked Adrenoleukodystrophy (X-ALD) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 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 X-linked Adrenoleukodystrophy (X-ALD) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

X-linked Adrenoleukodystrophy (X-ALD) Overview

X-linked adrenoleukodystrophy (X-ALD) is the most common peroxisomal disorder. It is distinguished by impaired peroxisomal beta-oxidation of very long-chain fatty acids, which is reduced to about 30% of control levels. Because of mutations in the ABCD1 gene, which encodes the peroxisomal membrane protein ALDP, VLCFA accumulates in the plasma and all tissues, including the brain's white matter, the spinal cord, and the adrenal cortex. In males with X-ALD, the clinical spectrum ranges from isolated adrenocortical insufficiency and slowly progressive myelopathy to devastating cerebral demyelination. By the age of 60, the majority of heterozygous females will have symptoms. In individual patients, the disease course remains unpredictable. Based on lipid inclusions in adrenal glands, brain and Schwann cells, testis, Schaumburg, and Powers suggested the term adrenoleukodystrophy for the disorder. Adrenocortical insufficiency can be the presenting symptom of X-ALD in boys and men years or even decades before neurological symptoms appear. X-ALD is a common cause of Addison's disease in boys and adult males, particularly when circulating adrenocortical auto is remarkably absent.

• The diagnosed incidence of X-linked adrenoleukodystrophy (X-ALD) varies between 1 to 1.5 cases per 15,000 to 20,000 live birth population in the USA.

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: 2021-2032

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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