DiGeorge Syndrome (DGS) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

DiGeorge Syndrome (DGS) Market Outlook

Thelansis’s “DiGeorge Syndrome (DGS) 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 DiGeorge Syndrome (DGS) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

DiGeorge Syndrome (DGS) Overview

DiGeorge syndrome (DGS) is a congenital disorder with a broad phenotypic presentation caused primarily by a microdeletion of chromosome 22 at 22q11.2. This mutation fails in the proper development of the pharyngeal pouches, which are responsible for the embryologic development of the middle and external ear, maxilla, palatine tonsils, mandible, thyroid, parathyroids, thymus, aortic arch, and cardiac outflow tract. DGS symptoms include cardiac anomalies, recurrent infections, abnormal facies, thymic hypoplasia or aplasia, cleft palate, developmental delay, and hypocalcemia. DGS symptoms include a thymus that is absent or hypoplastic, cardiac abnormalities, hypocalcemia, and parathyroid hypoplasia. The absence of thymic tissue, the organ responsible for T lymphocyte development, is the most concerning feature of DGS. A complete lack of the thymus, which is extremely rare and affects less than 1% of DGS patients, is associated with severe combined immunodeficiency (SCID). T-cells are specialized white blood cells specializing in immune functions such as destroying infected or malignant cells, acting as an integral part of the innate immune system by killing viruses, and assisting B-cells in maturing to produce immunoglobulins for stronger adaptive immunity. The degree of immunodeficiency in DGS patients can vary depending on the extent of thymic hypoplasia.

• The most common microdeletion syndrome is 22q11.2 microdeletion, which affects about 0.1 percent of fetuses. The rate of 22q11.2 microdeletion in live births is estimated to be 1 in 4000 to 6000.

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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