Glycogen Storage Disease (GSD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

 Glycogen storage diseases (GSDs) are hereditary disorders resulting from inborn errors in carbohydrate metabolism. These disorders lead to abnormal glycogen storage and are categorized as GSDs. They are assigned numerical classifications based on identifying the enzyme defect responsible for the condition. Clinical symptoms can manifest from infancy to adulthood. GSDs can arise due to the accumulation of toxic glycogen and the inability to convert glycogen into energy. However, in all cases, they result in an inability to utilize or store glycogen. There are various types of glycogen storage diseases, with the following being the most prevalent:

Glycogen storage disorders primarily affecting the liver encompass:

·       Glycogen synthase-2 deficiency (GSD type 0a)

·       Glucose-6-phosphatase deficiency (GSD type Ia)

·       Glucose-6-phosphate transporter deficiency (GSD type Ib)

·       Glycogen debrancher deficiency (GSD type III)

·       Glycogen branching enzyme deficiency (GSD type IV)

·       Liver phosphorylase deficiency (GSD type VI)

·       Phosphorylase kinase deficiency (GSD type IXa)

·       GLUT2 deficiency, also known as Fanconi-Bickel disease

Glycogen storage disorders primarily affecting skeletal muscles include:

·       Muscle phosphorylase deficiency (GSD type V)

·       Phosphofructokinase deficiency (GSD type VII)

·       Phosphoglycerate mutase deficiency (GSD type X)

·       Lactate dehydrogenase A deficiency (GSD type XI)

·       Aldolase A deficiency (GSD type XII)

·       Beta-enolase deficiency (GSD type XIII)

·       Phosphoglucomutase-1 deficiency (GSD type XIV)

Glycogen storage disorders affecting both skeletal and cardiac muscles include:

·       Lysosomal acid maltase deficiency (GSD type IIa)

·       Lysosome-associated membrane protein 2 deficiency (GSD type IIb)

·       Glycogenin-1 deficiency (GSD type XV)

·       Muscle glycogen synthase deficiency (GSD type 0b)

With early detection and proper management, most GSDs have a favorable prognosis. Effective dietary management reduces complications associated with hypoglycemia and has shown promise in ameliorating renal dysfunction in GSD type I patients. In rare instances, end-stage renal disease necessitating kidney transplantation may arise in GSD type Ib patients. For individuals with GLUT2 deficiency, an antiketogenic diet can significantly reduce liver size and glycogen content. In GSD type Ia patients, growth delays, short stature, renal dysfunction, hypertriglyceridemia, and hepatocellular carcinoma may occur. On the other hand, GSD type Ib can lead to recurrent bacterial infections due to neutropenia. GSD type IV is associated with progressive liver failure and cirrhosis. Patients with GSD type II may develop cardiomyopathy and limb-girdle dystrophy, while GSD type III often presents with hypertrophic cardiomyopathy as a classic complication.

 

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

 

KOLs insights of Glycogen Storage Disease (GSD) 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.

Glycogen Storage Disease (GSD) 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.

Thelansis Competitive Intelligence (CI) practice has been established based on a deep understanding of the pharma/biotech business environment to provide an optimized support system to all levels of the decision-making process. It enables business leaders in forward-thinking and proactive decision-making. Thelansis supports scientific and commercial teams in seamless CI support by creating an AI/ ML-based technology-driven platform that manages the data flow from primary and secondary sources.

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