Leigh Syndrome – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033
Leigh's syndrome, a rare inherited neurometabolic disorder affecting the central nervous system, typically emerges in infants aged three months to two years. However, it can infrequently arise during adolescence or early adulthood. Symptoms usually manifest before 12 months, but they may appear later in exceptional cases. Common initial symptoms include the loss of motor skills, hypotonia with poor head control, recurrent vomiting, and movement difficulties. Subsequently, pyramidal and extrapyramidal signs, nystagmus, breathing problems, ophthalmoplegia, and peripheral neuropathy may develop. Epilepsy is relatively uncommon. The syndrome causes problems with aerobic energy production, ranging from the pyruvate dehydrogenase complex to the oxidative phosphorylation pathway. Most mutations are in the nuclear genome, affecting genes related to the pyruvate dehydrogenase complex, respiratory complexes I or II, or proteins involved in respiratory complex IV assembly. Some individuals with Leigh syndrome carry mitochondrial DNA mutations, notably the 8993T>G or 8993T>C mutations in the MTATP6 gene. This subgroup, known as maternally inherited Leigh syndrome (MILS), has a high proportion of the mitochondrial DNA mutation. In contrast, a milder phenotype called NARP syndrome is associated with lower proportions of this mutation. In some cases, the genetic cause of Leigh syndrome remains unknown despite specific biochemical defects. Diagnosing the syndrome relies on brain imaging, which reveals characteristic brainstem and basal ganglia lesions, often with leucodystrophy and cerebral atrophy. Elevated lactate levels are consistently observed in cerebrospinal fluid and often in the blood. Biochemical investigations are used to identify the underlying energy production defect. Prenatal diagnosis is possible when a nuclear gene mutation is known, but it's more challenging when mitochondrial DNA is involved due to heteroplasmy. Currently, thiamine (Vitamin B1), oral sodium bicarbonate, or sodium citrate are commonly used treatments, although no specific approved treatment exists for Leigh's disease. The prognosis for Leigh's disease is generally poor. Individuals lacking mitochondrial complex IV activity and those with pyruvate dehydrogenase deficiency have the worst outlook, often succumbing within a few years. Those with partial deficiencies have a somewhat better prognosis, possibly surviving to 6 or 7 years of age and occasionally into their mid-teenage years.
Thelansis’s “Leigh Syndrome 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 Leigh
Syndrome treatment modalities options for eight major markets (USA, Germany,
France, Italy, Spain, UK, Japan, and China).
KOLs insights of Leigh Syndrome 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.
Leigh Syndrome 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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