Leukocyte Adhesion Deficiency Type II – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Leukocyte Adhesion Deficiency Type II Market Outlook

Thelansis’s “Leukocyte Adhesion Deficiency Type II 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 Leukocyte Adhesion Deficiency Type II treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Leukocyte Adhesion Deficiency Type II Overview

LAD II also recognized as a congenital disorder of glycosylation IIc (CDG IIc), is a rare congenital condition that disrupts the adhesion cascade's initial phase, specifically impeding selectin-mediated leukocyte tethering and rolling on the endothelium. Selectins adhere to glycoproteins or glycolipids adorned with fucosylated carbohydrate structures akin to sialyl Lewisx (sLex). LAD II leukocytes exhibit a near-complete lack of sLex expression, significantly impeding interactions with endothelial selectins and resulting in immunodeficiency. Furthermore, they do not express the H blood group antigen (Bombay phenotype) or the Lewis blood group antigens Lea and Leb, all of which are oligosaccharides containing a fucose unit, leading to the initial hypothesis that LAD II stems from a generalized defect in fucosylated structure biosynthesis. LAD II manifests with significantly elevated peripheral neutrophil counts, the cause of which remains elusive. One plausible explanation is the diminished migration of cells from the vasculature into tissues due to deficient selectin interactions. However, bone marrow leukocyte production increased in one patient, suggesting another potential cause for neutrophilia. Functionally, neutrophils in LAD II are deficient in rolling on endothelium and migrating into the skin, thus mechanistically contributing to the immunodeficiency seen in the condition. As of now, only seven cases of LAD II have been documented worldwide. Four patients of Arab descent were identified in Israel, residing in the same region, with three of them being distantly related. Additionally, a boy of Turkish heritage was diagnosed in Germany, and a boy of Brazilian origin was studied in the USA. The most recent case is a girl of Pakistani descent discovered in Scotland. 

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: 2023-2033

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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