DiGeorge Syndrome (DGS) — Market outlook, Epidemiology, Competitive Landscape, and Market Forecast Report — 2021 To 2032
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.
The competitive landscape of DiGeorge Syndrome (DGS) includes country-specific approved and pipeline therapies. Any asset/product-specific designation, review, and Accelerated Approval are tracked and supplemented with analyst commentary.
KOLs insights of DiGeorge Syndrome (DGS) 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.
DiGeorge Syndrome (DGS) Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard), which Data Inputs with sourcing, Market Event, Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.
S. No Asset Company Stage
1 Zygel™ Zynerba Pharmaceuticals, Inc. Phase 1
2 Cultured Thymus Tissue Implantation (CTTI) Enzyvant Therapeutics GmBH Phase 2
3 NB-001 Nobias Therapeutics, Inc. Phase 2
