Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i) Market Outlook

Thelansis’s “Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i) 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 2i treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i) Overview

Limb-girdle muscular dystrophies (LGMDs) encompass a set of muscle disorders with varying inheritance patterns, either autosomal dominant (referred to as LGMD type 1) or autosomal recessive (known as LGMD type 2). The fukutin-related protein gene (FKRP; OMIM 606596) plays a significant role in developing both LGMD2I and congenital muscular dystrophy 1C. FKRP transcripts are predominantly expressed in skeletal muscle, the placenta, and the heart. Altered α-dystroglycan protein expression patterns have been observed in muscle biopsies of LGMD2I patients, indirectly suggesting that FKRP, a potential glycosyltransferase, is necessary for post-translational dystroglycan modification. Diagnosis typically concerns the clinical presentation, characterized by proximal muscle wasting and weakness, particularly in the lower limbs. The primary diagnostic criterion involves the identification of two pathogenic variants in the FKRP gene. Creatine kinase levels are usually elevated, exceeding 1,000, and can drop to undetectable levels following strenuous exercise, possibly resulting in myoglobinuria. Muscle biopsies reveal dystrophic changes, and staining for alpha-dystroglycan glycosylation using VIA4 and IIH6 antibodies demonstrates glycosylation loss. The decline in motor function and respiratory limitations significantly impact the quality of life. Life expectancy remains uncertain but typically normal in individuals homozygous for the c.826C>A mutation. Patients with compound heterozygosity for the mutation will likely have a reduced life expectancy due to early-age invasive ventilation and ensuing complications. Currently, no FDA-approved treatments are specifically for limb-girdle muscular dystrophy type 2i (LGMD2I). Existing interventions focus solely on providing supportive care.

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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