GM1 Gangliosidosis – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

GM1 Gangliosidosis Market Outlook

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

GM1 Gangliosidosis Overview

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.

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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