Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033 thelansis.com

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.

Thelansis’s “Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033″ 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 (LGMD2i) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

KOLs insights of Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i) across 8 MM market from the centre of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.
Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i) Market Forecast Patient Based Forecast Model (MS. Excel Based Automated Dashboard), which 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.
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