Gorlin Syndrome – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

Gorlin Syndrome Market Outlook

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

Gorlin Syndrome Overview

Gorlin syndrome, also known as Gorlin-Goltz syndrome, nevoid basal cell carcinoma syndrome (BCNS), or basal cell nevus syndrome, is an autosomal dominant familial cancer syndrome. It manifests with numerous basal cell carcinomas (BCCs) and is accompanied by skeletal, ophthalmologic, and neurologic abnormalities. The syndrome gives rise to multiple neoplasms that initiate during childhood. The underlying cause of basal cell nevus syndrome is mutations within the patched (PTCH) gene. This gene encodes a transmembrane receptor responsible for recognizing sonic hedgehog signaling proteins. The syndrome demonstrates a high degree of penetrance while expressing variability in its presentation. Around 20% to 30% of BCNS cases are due to de novo mutations. Individuals harboring one mutated copy of the PTCH1 gene in each cell can exhibit Gorlin syndrome features early in life, including skeletal irregularities and macrocephaly. For basal cell carcinomas and other tumors to emerge, a mutation must also affect the second copy of the PTCH1 gene in specific cells during the person's lifetime. Individuals with one PTCH1 gene mutation will eventually acquire a second mutation in certain cells, ultimately developing diverse tumor types. Gorlin syndrome presents a range of symptoms and signals termed "indicators." These include:

1. Basal cell carcinomas (BCCs) are a form of skin cancer appearing on the face, hands, and back of the neck
2. Odontogenic keratocystic tumors (OKCs) benign cysts found in the jawbone
3. Small depressions or pits on the palms of the hands and the soles of the feet

Currently, there exists no specific treatment or cure for Gorlin syndrome itself. Management of this syndrome focuses on preventing, handling, and removing tumors and cysts stemming from the condition. Various methods are available to eliminate the tumors and cysts associated with Gorlin syndrome, such as cryotherapy (freezing), laser treatments, and curettage (scraping).

• Gorlin syndrome is a rare genetic disorder that affects roughly 11,000 individuals in the United States.

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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