GM2 Gangliosidosis – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033
GM2 gangliosidosis is a rare, inherited disorder that causes progressive damage to the nervous system. It is caused by a deficiency of the enzyme beta-hexosaminidase, which breaks down a type of fat called GM2 ganglioside. When GM2 ganglioside accumulates in cells, it damages neurons and other brain and spinal cord cells. GM2 gangliosidosis is divided into three clinical subtypes based on the age at onset:
·
Type 1 (Tay-Sachs disease) is the most common
and severe form, with onset in the first few months of life. Children with type
1 GM2 gangliosidosis typically experience developmental delays, loss of motor
skills, seizures, and vision and hearing loss. They usually die by the age of
4.
·
Type 2 (Sandhoff disease) is less common than
type 1 and has a later onset, typically between the ages of 1 and 3. Children
with type 2 GM2 gangliosidosis experience similar symptoms to those with type
1, but the progression is slower. They may live into their teenage years or
early adulthood.
·
Type 3 (juvenile GM2 gangliosidosis) is the
rarest and mildest form of the disease, with onset in childhood or adolescence.
Children with type 3 GM2 gangliosidosis may experience various neurological
symptoms, such as ataxia, dysarthria, and cognitive decline. The progression of
type 3 GM2 gangliosidosis is highly variable, and some people may live into
adulthood.
GM2
gangliosidosis is an autosomal recessive disorder, meaning both parents must
carry a defective gene for the child to be affected. The incidence of GM2
gangliosidosis in the general population is estimated to be 1-9 cases per
100,000 live births. However, the incidence is higher in certain populations,
such as Ashkenazi Jews and Cajuns. There is no cure for GM2 gangliosidosis, but
treatment can help to relieve symptoms and improve the quality of life for
affected individuals. Treatment options may include supportive care, feeding
assistance, pain management, and therapies to improve speech, language, and
motor skills.
Thelansis’s
“GM2 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 GM2
Gangliosidosis treatment modalities options for eight major markets (USA,
Germany, France, Italy, Spain, UK, Japan, and China).
KOLs insights of GM2 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.
GM2
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.
Tags: GM2
Gangliosidosis, GM2 Gangliosidosis market outlook, GM2
Gangliosidosis competitive landscape, GM2
Gangliosidosis market forecast, Thelansis, Primary
market research, KOL insights, Competitive Intelligence (CI)
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