Limb-Girdle Muscular Dystrophy Type 1 – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Limb-Girdle Muscular Dystrophy Type 1 Market Outlook

Thelansis’s “Limb-Girdle Muscular Dystrophy Type 1 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 Limb-Girdle Muscular Dystrophy Type 1 treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Limb-Girdle Muscular Dystrophy Type 1 Overview

Limb-Girdle Muscular Dystrophy Type 1 (LGMD1A, LGMD1B, LGMD1C, & Others (LGMD1D-H)) includes forms of the disorder that have an inheritance pattern called autosomal dominant. Majorly the LGMD1B comprises more than 65% of the total Limb-girdle muscular dystrophy type 1, followed by LGMD1C, LGMD1A, & Others type. The autosomal dominant forms of LGMD are less common than the autosomal recessive forms and appear later in life. In many cases, autosomal dominant LGMD progresses more slowly than autosomal recessive LGMD and has variable symptoms, even among members of the same family. Each gene mutation can cause a wide range of symptoms. LGMD1A can appear at any age, from adolescence to adulthood. This type of LGMD is distinguished by proximal muscle weakness, which is sometimes accompanied by slurred speech (dysarthria) and an abnormally tight Achilles tendon. LGMD1B causes proximal muscle weakness that gradually worsens. Affected individuals may also experience calf muscle hypertrophy and mild elbow or Achilles tendon contractures. LGMD1C is characterized by cramping muscle pain after exercise, mild to moderate proximal muscle weakness, and overgrowth of the calf muscle. LGMD1D-H short form is characterized by progressive muscle weakness that affects the hip-girdle area before spreading to the limb-girdle area.

• The incidence of Limb-girdle muscular dystrophy type 1 varies from 3 to 4 cases per million population in the USA.

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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