Charcot-Marie-Tooth Disease Type 1A (CMT1A) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

Charcot-Marie-Tooth Disease Type 1A (CMT1A) Market Outlook

Thelansis’s “Charcot-Marie-Tooth Disease Type 1A (CMT1A) 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 Charcot-Marie-Tooth Disease Type 1A treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Charcot-Marie-Tooth Disease Type 1A (CMT1A) Overview

Charcot-Marie-Tooth disease type 1 (CMT1) is a collection of demyelinating peripheral neuropathies inherited autosomal dominant. It is characterized by weakness and wasting of muscles in the distal regions of the body, loss of sensation, foot deformities, and reduced nerve conduction velocity. The age at which CMT1 manifests can vary significantly, ranging from infancy to the fourth decade or later. Generally, symptoms appear between the first and second decade of life, although the full clinical presentation may emerge later. The severity of the disease can range from very mild cases that go unnoticed to substantial weakness and disability. Affected individuals typically experience weakness in the distal muscles, symmetric wasting (mainly in the peroneal muscles), and diminished or absent tendon reflexes. Sensory deficits, including impaired position sense, vibration sense, and pain/temperature sensation, are common in the feet and may later affect the hands. Pes cavus (high arches) or planus (flat feet) with hammer toes are often observed from childhood. Scoliosis of varying degrees may develop during adolescence. CMT1A is caused by duplications of a 1.4 Mb segment or point mutations in the PMP22 gene located on chromosome 17p12. Diagnosis of CMT1 is based on progressive motor and sensory neuropathy, slow nerve conduction velocity (NCV) with motor NCV less than 38 m/s in the upper limbs, and positive family history (although sporadic cases can occur due to new mutations). Genetic testing is the definitive method for diagnosis. Differential diagnosis should consider other genetic neuropathies, particularly X-linked CMT, autosomal dominant CMT2, CMT4, and hereditary neuropathy with liability to pressure palsies. CMT1 progresses slowly and affected individuals experience long periods of stability without noticeable deterioration. Most patients maintain their ability to walk throughout their lives and have an average life expectancy.

• CMT1A was the most common subtype of CMT, with a prevalence rate of 1 per 5000.

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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