New York, USA, July 24, 2024 (GLOBE NEWSWIRE) -- DelveInsight’s report includes a comprehensive understanding of current treatment practices, Leigh syndrome emerging drugs, market share of individual therapies, and current and forecasted Leigh syndrome market size from 2020 to 2034, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan]. Discover which therapies are expected to grab Leigh syndrome market share @ Leigh syndrome is a rare genetic disorder affecting neurometabolism. It is marked by the deterioration of the central nervous system, including the brain, spinal cord, and optic nerve.
Typically, Leigh syndrome is passed down as an autosomal recessive trait, but it can also be inherited through X-linked recessive or maternal transmission due to mitochondrial DNA mutations. The symptoms of Leigh syndrome often worsen quickly. Initial signs may include difficulty sucking, loss of head control and motor skills, decreased appetite, vomiting, and seizures.
As the disease advances, it may cause muscle weakness, reduced muscle tone, spasticity, movement disorders, cerebellar ataxia, and peripheral neuropathy. Complications can affect respiratory, heart, and kidney functions. Diagnosing Leigh syndrome involves a detailed clinical evaluation and various specialized tests, particularly advanced imaging methods.
MRI or CT scans of the brain can reveal abnormalities in specific areas such as the basal ganglia, brain stem, and gray matter. The Leigh syndrome epidemiology section provides insights into the historical and current Leigh syndrome patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.
The proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Download the report to understand which factors are driving Leigh syndrome epidemiology trends @ There is no particular treatment for Leigh syndrome, and generally, specific therapies for mitochondrial disorders in children are not widely accessible. Symptomatic treatment aims to manage symptoms and enhance energy levels by boosting ATP production and reducing lactate levels. This is achieved through the elimination of harmful metabolites and the use of oxygen radical scavengers.
Antioxidants are frequently used in treatment. Symptomatic treatments for Leigh syndrome include supplementation with B vitamins like thiamine, antiseizure medications, physiotherapy, and pharmacotherapy. Various substances, commonly known as 'nutraceuticals' because they are derived from everyday foods, have been suggested for treating LS or other mitochondrial disorders.
These substances include , among others. Often, patients receive a combination of these compounds as part of their treatment. Symptomatic treatments, such as antiseizure medication to manage seizures, are crucial components of care.
Controlling seizures without additional side effects can greatly enhance quality of life. Addressing neuromuscular issues through physiotherapy and medication, using proper feeding techniques to prevent malnutrition and aspiration, and promptly treating fever and infections are also key aspects of treatment aimed at improving quality of life. Learn more about the FDA-approved drugs for Leigh syndrome @ The market has a promising outlook with some of the potential emerging therapies for the treatment of Leigh syndrome.
Some of the drugs in the pipeline include , among others. , derived from alpha tocotrienol, a form of vitamin E, is the resulting compound after oxidation. It acts on the enzyme 15-lipoxygenase, crucial in managing oxidative stress and inflammation across various tissues.
By inhibiting this enzyme, it reduces inflammation and oxidative stress, thereby enhancing cellular health. In October 2019, the company obtained the assets of BioElectron, which included EPI-743. Back in June 2014, the FDA granted orphan drug designation for Vatiquinone for treating Leigh Syndrome.
Presently, Vatiquinone is undergoing Phase III trials to evaluate its safety in individuals with inherited mitochondrial disorders who were previously exposed to it in earlier clinical studies or treatment plans sponsored by PTC/BioElectron. This trial encompasses patients with Leigh syndrome as well. is in the developmental stages for treating mitochondrial disease, particularly targeting insufficient activity in mitochondrial protein complex I, which leads to a deficiency in energy conversion and manifests as various neurologic complications in diseases like Leigh syndrome, MELAS, or LHON.
This drug candidate was identified for its ability to enhance mitochondrial function in cells of patients with mitochondrial Leigh syndrome. NV354, which can penetrate the brain, is a product of innovation wherein the body's own energy substrate, succinate, is made available in cells using prodrug technology. Preclinical development for this program has been finalized, and in April 2023, the drug received in the US for mitochondrial disease treatment.
Additionally, in December 2023, Abliva secured for NV354 from the European Commission specifically for treating Leigh syndrome. The anticipated launch of these emerging therapies are poised to transform the Leigh syndrome market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the Leigh syndrome market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.
To know more about Leigh syndrome clinical trials, visit @ The Leigh syndrome market dynamics are anticipated to change in the coming years. The Leigh syndrome market is primarily driven by , , and a . Enhanced funding from both governmental and non-governmental organizations for mitochondrial disorder studies has accelerated the .
Additionally, the has facilitated greater collaboration among researchers, healthcare providers, and pharmaceutical companies, fostering a more . The expanding and the to improve clinical trial methodologies further contribute to the market's growth potential. Furthermore, many potential therapies are being investigated for the treatment of Leigh syndrome, and it is safe to predict that the treatment space will significantly impact the Leigh syndrome market during the forecast period.
Moreover, the with improved efficacy and a further are expected to drive the growth of the Leigh syndrome market in the 7MM. However, several factors may impede the growth of the Leigh syndrome market. The , stemming from its genetic heterogeneity and variable clinical presentations, further .
Additionally, there are substantial challenges related to , as well as a that can inform clinical trial design. for rare diseases also pose significant obstacles, making it difficult for new treatments to reach the market and patients who desperately need them. Moreover, Leigh syndrome treatment poses a and disrupts patients’ overall well-being and QOL.
Furthermore, Leigh syndrome market growth may be offset by , , , and a . In addition, the about the disease may also impact Leigh syndrome market growth. Discover more about Leigh syndrome drugs in development @ report delivers an in-depth understanding of the disease, historical and forecasted Leigh syndrome epidemiology in the 7MM, i.
e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Leigh syndrome companies, including among others.
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report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Tay-Sachs disease companies, including among others. DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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