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Recent News Stories Shows How The United Kingdom Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics Market Products Are Surviving In The Global Industry To 2028?

03-03-2023 06:27 PM CET | Health & Medicine

Press release from: Prudent Markets

UK Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics market

UK Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics market

Report Overview

The UK Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics market size stood at around USD xx billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Oculopharyngeal Muscular Dystrophy (OPMD) is a rare, adult-onset, progressive myopathy characterized by progressive eyelid ptosis, ophthalmoplegia, dysphagia, dysarthria, and proximal limb weakness. OPMD is a genetic muscle disorder that mainly affects the muscle of the upper eyelids and throat. OPMD is caused by an expansion in the polyalanine tract in the PABPN1 gene (14q11.2), which leads to overexpression of a mutant protein, polyadenylate-binding protein 2, and consequently to the accumulation of nuclear aggregates in the muscles.

Disease onset occurs in the fifth to sixth decade of life. Early signs include ptosis, ophthalmoplegia without diplopia, limb weakness, dysarthria, and dysphagia. Symptoms usually begin after the age of 45 years, and ptosis is the most common presenting feature. In several cases, limb weakness has preceded dysphagia. In 5-10% of patients, the disease is more severe, with ptosis and dysphagia presenting before the age of 45 and incapacitating distal leg weakness occurring before the age of 60. The manifestations of autosomal recessive OPMD usually present later (after the age of 60) than those of the autosomal dominant form.

OPMD is a rare disorder that affects males and females in equal numbers. The OPMD is among the 30 inherited diseases declared by WHO and its results in the weakening and worsening of muscles. OPMD affects less than 5 in 10,000 people in the EU. According to a study, in Europe, the estimated prevalence of oculopharyngeal muscular dystrophy is 1 in 100,000 people. The analysis demonstrates the phenotypic and genotypic characteristics of 31 patients with OPMD in the UK. Ptosis was the first reported symptom in two-thirds of the patients, and half of the subjects studied had evidence of ophthalmoplegia. The PABP2 analysis is a reliable non-invasive diagnostic test for OPMD in the UK population.

Market Growth Drivers

The major driving factor involved in the OPMD therapeutics market is the increased incidence and prevalence of oculopharyngeal muscular dystrophy in the middle-aged population. Conventional therapy mainly focused on symptomatic treatment of Oculopharyngeal Muscular Dystrophy and having lots of off-target effects, this factor providing the additional opportunity for key players present in the OPMD treatment market to expand their research and development activities and expected to drive the growth of oculopharyngeal muscular dystrophy treatment market during the forecast period.

Additionally, the introduction of new disease-modifying and novel drug therapies, increased drug development activities are expected to drive the oculopharyngeal muscular dystrophy treatment market. Furthermore, gene therapies such as mutation suppression, exon skipping are also anticipated to push the OPMD treatment market during the forecast period.

Market Restraints

On the contrary, the high cost of the novel therapies and lack of awareness among individuals are some factors that are expected to hamper the growth of the oculopharyngeal muscular dystrophy treatment market during the forecast period.

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Top Major Players

Increased demand for efficacious therapies among patients, rising investment in research and development activities, and introduction of novel disease therapies like RNA replacement strategy, and mutation suppression are anticipated to make the OPMD therapeutics market highly competitive. As of now, 'Seelos Therapeutics' emerged as a major key player involved in the Oculopharyngeal Muscular Dystrophy treatment market, had agreed with Bioblast Pharma Ltd., to acquire all development and commercial rights to Bioblast's proprietary drug named Cabaletta (trehalose).

The United Kingdom Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics Market 2023 Report makes available the current and forthcoming technical and financial details of the industry. It is one of the most comprehensive and important additions to the Prudent Markets archive of market research studies. It offers detailed research and analysis of key aspects of the global United Kingdom Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics market. This report explores all the key factors affecting the growth of the global United Kingdom Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics market, including demand-supply scenario, pricing structure, profit margins, production, and value chain analysis.

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Market Segmentation

By Diagnosis:

Based on the diagnosis, the OPMD therapeutics market can be bifurcated into

Blood tests
Enzyme assay
Genetic testing
Electromyography
Biopsy
Others

The diagnosis of OPMD can be confirmed through commercially available blood tests that detect the specific genetic abnormality in the PABPN1 gene, known as a repeat expansion, that is associated with OPMD (i.e., mutation of the PABPN1 gene).

By Therapeutics:

Based on therapeutics, the OPMD therapeutics market can be segmented into

Non-steroidal anti-inflammatory drugs (NSAIDs)
Corticosteroids
ACE inhibitors
Gene Replacement Therapy
Molecular Therapy
Stem Cell Research
Mutation Suppression
Exon Skipping
Others

Corticosteroids, such as prednisone and deflazacort (Emflaza), which can help muscle strength and delay the progression of OPMD are commonly preferred. Additionally, novel gene therapies such as CRISPR /Cas9, RNA Replacement strategy to develop promising therapy for Oculopharyngeal Muscular Dystrophy.

By Route of Administration:

Based on the route of administration, the OPMD therapeutics market can be segmented into

Oral
Intravenous (IV)
Others

As per the latest report, there is currently only one treatment for people with Oculopharyngeal Muscular Dystrophy available on the UK's National Health Service (NHS); Cabaletta (trehalose), which is delivered intravenously once a week. Therefore, the intravenous type of the route of administration segment is anticipated to account for the majority of the revenue share during the forecast period.

Top Major Players

Increased demand for efficacious therapies among patients, rising investment in research and development activities, and introduction of novel disease therapies like RNA replacement strategy, and mutation suppression are anticipated to make the OPMD therapeutics market highly competitive. As of now, 'Seelos Therapeutics' emerged as a major key player involved in the Oculopharyngeal Muscular Dystrophy treatment market, had agreed with Bioblast Pharma Ltd., to acquire all development and commercial rights to Bioblast's proprietary drug named Cabaletta (trehalose).

Prudent Markets provides attractive discounts that fit your needs. Customization of the reports as per your requirement is also offered. Get in touch with our sales team, who will guarantee you a report that suits your needs.

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The report covers the competitive analysis of the market. As the demand is driven by a buyer's paying capacity and the rate of item development, the report shows the important regions that will direct growth. This section exclusively shares insight into the budget reports of big-league members of the market helping key players and new entrants understand the potential of investments in the Global United Kingdom Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics Market. It can be better employed by both traditional and new players in the industry for complete know-how of the market.

Strategic Points Covered in Table of Content of Global United Kingdom Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics Market:

Chapter 1: Introduction, market driving force product Objective of Study and Research Scope the United Kingdom Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics market
Chapter 2: Exclusive Summary - the basic information of the United Kingdom Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics Market.
Chapter 3: Displaying the Market Dynamics- Drivers, Trends and Challenges & Opportunities of the United Kingdom Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics
Chapter 4: Presenting the United Kingdom Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics Market Factor Analysis, Porters Five Forces, Supply/Value Chain, PESTEL analysis, Market Entropy, Patent/Trademark Analysis.
Chapter 5: Displaying the by Type, End User and Region/Country
Chapter 6: Evaluating the leading manufacturers of the United Kingdom Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics market which consists of its Competitive Landscape, Peer Group Analysis, BCG Matrix & Company Profile
Chapter 7: To evaluate the market by segments, by countries and by Manufacturers/Company with revenue share and sales by key countries in these various regions
Chapter 8 & 9: Displaying the Appendix, Methodology and Data Source

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Free Customization on the basis of client requirements on Immediate purchase:
1- Free country-level breakdown of any 5 countries of your interest.
2- Competitive breakdown of segment revenue by market players.

Customization of the Report:
This report can be customized to meet the client's requirements. Please connect with our sales team (sales@prudentmarkets.com), who will ensure that you get a report that suits your needs. You can also get in touch with our executives on +91 83560 50278 || USA/Canada(Toll Free): 1800-601-6071 to share your research requirements.

Get ready to Recognize the pros and cons of the regulatory framework, local reforms, and its effect on the Industry. Understand how the Leaders in Intelligent Network are keeping themselves one stage forward with our most up-to-date survey analysis.

In conclusion, the United Kingdom Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics Market report is a genuine source for accessing the research data which is projected to exponentially grow your business. The report provides information such as economic scenarios, benefits, limits, trends, market growth rates, and figures. SWOT analysis and PESTLE analysis is also incorporated in the report.

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