Proteus Syndrome – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Proteus Syndrome Market Outlook

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

Proteus Syndrome Overview

Proteus syndrome (PS) is an exceptionally rare and intricate disorder characterized by excessive growth in the skeletal, skin, adipose tissue, and central nervous system. Causal mutations for PS have been identified in two components of the phosphatidylinositol 3-kinase (PI3K)-AKT signaling pathway: PTEN, linking PS to the PTEN Hamartoma Tumor Syndrome (PTHS), and AKT1. The AKT1 mutation is typically a somatic mosaic mutation, found in 1-47% of cases. Newborns with PS usually appear normal at birth. Symptoms typically manifest between 6 and 18 months of age and often involve asymmetric overgrowth, primarily in the hands or feet. Macrodactyly, characterized by abnormally large digits, is the most common initial symptom, often accompanied by hemihypertrophy. Skeletal overgrowth can progress rapidly, resulting in irregular, distorted, and calcified growth in the tubular bones of the limbs, skull, and vertebral bodies. Diagnosing PS relies on specific clinical criteria. To establish a diagnosis, patients must meet the general criteria, which include the mosaic distribution of lesions, sporadic occurrence, and a progressive course. Additionally, patients must meet either one of the specific category A criteria (CCTN), two category B criteria (asymmetric disproportionate overgrowth, linear epidermal nevus, specific tumors before the 2nd decade), or three category C criteria (vascular malformations, dysregulated adipose tissue, distinct facial phenotype). Failure to meet these criteria may result in a diagnosis of Proteus-like syndrome. Molecular genetic testing can confirm the diagnosis. Differential diagnoses for PS encompass Klippel-Trenaunay-Weber syndrome, hemihypertrophy, Ollier disease, macrodactyly, Maffucci syndrome, CLOVE syndrome, neurofibromatosis type 1, and other PTEN Hamartoma Tumor Syndrome disorders. Managing PS requires a multidisciplinary approach. Interventions to control the excessive growth of tubular bones may include epiphysiostasis, epiphysiodesis, and, in severe cases, amputation.

• Proteus syndrome is a rare condition with an incidence of less than 1 in a million people worldwide.

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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