Thelansis

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 infanti

 GM1 gangliosidosis is a rare lysosomal storage disorder characterized biochemically by deficient beta-galactosidase activity and clinically by a wide range of variable ophthalmological, neurovisceral and dysmorphic features. Prognosis depends on the GM1 gangliosidosis type and is extremely poor in the severe infantile form and variable in the chronic adult form of the disease. ·        According to Thelansis, the overall prevalence at birth of GM1 gangliosidosis is estimated to be approximately 1.5 to 2.1 per 100,000 live births. 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, German

  GM1 gangliosidosis is a rare lysosomal storage disorder characterised biochemically by deficient beta-galactosidase activity and clinically by a wide range of variable neurovisceral, ophthalmological and dysmorphic features. Prognosis depends on the type of GM1 gangliosidosis and is extremely poor in the severe infantile form and variable in the chronic adult form of the disease. ·        According to Thelansis, the overall prevalence at birth of GM1 gangliosidosis is estimated to be approximately 1.5 to 2.1 per 100,000 live births. ·        Lack of adequate medical treatment is the unmet need in this space; symptomatic treatment for some neurologic signs and symptoms is available but does not significantly alter the condition’s progression. For example, anticonvulsants may initially control seizures. ·        Few companies like Passage Bio, Axovant Sciences,  etc., are also developing assets in the pre-clinical stages.   The competitive landscape of GM1 Gangliosidosis incl