Primary Mitochondrial Disease – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033 thelansis.com

Primary Mitochondrial Disease (PMM) encompasses a diverse set of conditions marked by abnormalities in mitochondria, either functional or structural, resulting in disruptions in cellular energy production, the generation of reactive oxygen species and free radicals, and the impairment of various intracellular metabolic processes. These dysfunctions can lead to dysfunction in one or multiple organs. PMM originates from inherited genetic mutations affecting either mitochondrial or nuclear DNA, with over 350 genes implicated in its development. These mutations disrupt normal mitochondrial function, which is responsible for most cellular energy production. Chronic Progressive External Ophthalmoplegia (CPEO) is one of the most common manifestations of PMM. It manifests with symptoms like drooping eyelids (ptosis), muscle paralysis governing eye movement, and sometimes limb weakness. The onset of PMM can occur at any age, with patients experiencing severe, widespread muscle issues generally appearing early in life. Conversely, individuals with milder symptoms or muscle-specific problems tend to present later. Typically, the earlier the disease emerges, such as in infancy or early childhood, the more severe the mitochondrial disorder tends to be. MERRF (Myoclonic epilepsy with ragged-red fibers) is another primary mitochondrial disease characterized by involuntary muscle twitches, coordination difficulties, epilepsy, and potential impacts on various bodily functions. Microscopic examination of muscle tissue reveals distinct changes. This disorder is triggered by mutations in mitochondrial DNA, particularly the m.8344A>G mutation. Leigh Syndrome, a devastating inherited primary mitochondrial disease, primarily affects children early on, resulting in severe, progressive disability or childhood mortality. Symptoms typically manifest between ages one and two, often during episodes of illness or infection. Following onset, the condition deteriorates in episodes and frequently leads to early death, with patients reaching a median age of 2.4 years. The diagnosis of PMM hinges on the presence of associated symptoms and requires confirmation through specialized tests, including molecular genetic testing for PMM-related genes, muscle function assessments through exercise testing, lactic acid level measurements in blood or cerebrospinal fluid, metabolic enzyme activity assessments in blood, electromyography and nerve conduction studies to gauge muscle and nerve activity, muscle biopsies, and brain imaging techniques such as computed tomography (CT) scans or magnetic resonance imaging (MRI). Notably, there are no approved pharmaceutical treatments for primary mitochondrial disease, except for a specific hereditary mitochondrial eye disorder called LHON (Leber’s Hereditary Optic Neuropathy), where Raxone® (idebenone) has gained approval in Europe and Israel. Nevertheless, significant unmet medical needs remain.
• Approximately 125 people per million are estimated to be affected by primary mitochondrial disease.

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

KOLs insights of Primary Mitochondrial Disease 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.

Primary Mitochondrial Disease 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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