GM1 Gangliosidosis – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

GM1 gangliosidosis is a rare inherited lysosomal storage disorder characterized by a deficiency of the enzyme beta-galactosidase. This enzyme breaks down gangliosides, a type of lipid found in cell membranes. When beta-galactosidase is deficient, gangliosides accumulate in cells, particularly in the nervous system. There are three clinical subtypes of GM1 gangliosidosis, classified by age of symptom onset:

1.       Infantile (type 1): This is the most common and severe form of GM1 gangliosidosis, with symptom onset by six months and death by two to four years. Affected infants typically appear normal at birth but soon develop neurological symptoms such as hypotonia (weakness), seizures, and developmental delay.

2.       Late infantile/juvenile (type 2): This form of GM1 gangliosidosis presents as early as 7 months and as late as five years of age, with death occurring in mid-childhood to early adulthood. Affected children typically have a milder disease course than those with infantile GM1 but still experience a progressive neurological decline.

3.       Adult/chronic (type 3): This is the least severe form of GM1 gangliosidosis, with slower disease progression and symptom onset from early childhood to the late teens. Affected individuals may experience various symptoms, including neurological problems, psychiatric symptoms, and visceral (internal organ) problems.

The prognosis for GM1 gangliosidosis depends on the subtype. Patients with infantile GM1 have the poorest prognosis, most dying by two to four years of age. Patients with late infantile/juvenile GM1 have a longer life expectancy but still experience progressive neurological decline. Adult/chronic GM1 patients have the best prognosis but may still experience significant disability. There is no cure for GM1 gangliosidosis, but there are treatments that can help manage the symptoms and improve the quality of life for affected individuals. Treatment options include anticonvulsants to control seizures, gastrostomy tubes to ensure proper nutrition and hydration, and procedures to maintain an open airway. Diagnosis of GM1 gangliosidosis is typically made by measuring beta-galactosidase enzyme activity in white blood cells or skin cells. Genetic testing can also be used to confirm the diagnosis. Current treatment options for GM1 gangliosidosis are limited to palliative and supportive care. However, several promising new therapies are developing, including gene and enzyme replacement therapies. These therapies can potentially slow the progression of the disease and improve the quality of life for affected individuals.

·       According to Thelansis, the overall prevalence of GM1 gangliosidosis at birth is approximately 1.5 to 2.1 per 100,000 live births.

 

Thelansis’s “GM1 Gangliosidosis 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 GM1 Gangliosidosis treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

 

KOLs insights of GM1 Gangliosidosis 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.

GM1 Gangliosidosis 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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Tags: GM1 Gangliosidosis, GM1 Gangliosidosis market outlook, GM1 Gangliosidosis competitive landscape, GM1 Gangliosidosis market forecast, Thelansis, Primary market research, KOL insights, Competitive Intelligence (CI)