근긴장성이영양증 시장 : 시장 인사이트, 역학, 시장 예측(-2032년)
Myotonic Dystrophy - Market Insight, Epidemiology And Market Forecast - 2032
주요 7개국의 근긴장성이영양증 시장 규모는 2021년에 8,000만 달러로 평가되었습니다. 이 시장은 앞으로도 한층 더 확대될 것으로 예측됩니다.
주요 7개국의 근긴장성이영양증(Myotonic Dystrophy) 시장에 대해 조사했으며, 시장 개요와 함께 역학, 환자 동향, 새로운 치료법, 2032년까지 시장 규모 예측 및 의료 미충족 요구 등의 정보를 제공합니다.
DelveInsight's 'Myotonic Dystrophy (DM) - Market Insights, Epidemiology, and Market Forecast - 2032' report delivers an in-depth understanding of the historical and forecasted epidemiology as well as the market trends of DM in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.
The myotonic dystrophy (DM) market report provides current treatment practices, emerging drugs, market share of the individual therapies, and current and forecasted myotonic dystrophy (DM) market size from 2019 to 2032, segmented by 7MM. The report also covers the current myotonic dystrophy (DM) treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying market potential.
Study period: 2019-2032.
According to the National Organization for Rare Disorders (NORD), myotonic dystrophy (DM) refers to a group of two rare genetic (autosomal dominant) disorders of muscle affecting multiple systems of the body; characterized by a clinical triad of progressive weakness, myotonia, and early-onset cataracts. DM is a type of muscular dystrophy. Muscular dystrophies are characterized by weakness and degeneration of various voluntary muscles of the body. Specific abnormalities characterize each disorder (e.g., variation of muscle fiber size, muscle fiber necrosis, scar tissue formation, and inflammation) in muscle biopsy from patients. There are two main types of DM- DM type 1 (DM1) and DM type 2 (DM2). DM1 is also known as Steinert disease, named after Dr. Steinert, who, along with colleagues, first described the classic form in the medical literature in 1909. DM2 is also known as Ricker syndrome or proximal DM or PROMM.
A change or alteration causes DM1 in the myotonic dystrophy protein kinase (DMPK) gene. DM2 is caused by a change or alteration in the nucleic acid-binding protein (CNBP) gene, also called the ZNF9 gene. It is caused when a certain segment of DNA at the end of a gene is abnormally repeated many times, forming an unstable region in the gene, and is called a triplet repeat expansion. Unaffected individuals have a small number of repeats (up to about 35) in this region, but in affected individuals, the number can be much higher- rising to several thousand in congenitally affected population; DM1 is estimated to affect about 1 in 8,000-20,000 people in the general population. However, the prevalence of DM1 and DM2 vary greatly among different countries and ethnic groups.
Diagnosis of DM may be suspected based upon a thorough clinical evaluation, a detailed patient and family history, and identifying characteristic physical findings. A family history of muscle weakness and myotonia strongly indicates a DM diagnosis. Several laboratory tests can clarify the clinical diagnosis of DM, including blood tests, electromyography (EMG), magnetic resonance imaging (MRI), muscle biopsy, and genetic testing. Though different diagnostic options are available, the definitive test for DM is a genetic test.
Currently, no approved treatment offers a permanent cure for DM, but researchers are studying ways to help people with these disorders. Current treatment is directed toward the specific symptoms that are apparent in each individual. Pharmacological management of DM involves the usage of pain medications like nonsteroidal anti-inflammatories (NSAIDs), gabapentin, tricyclic antidepressants, mexiletine, and low-doses of glucocorticoids such as prednisone. Surgical procedures are opted for removing cataracts if they affect vision. However, there have been reports that cataracts can recur after removal. Some people with breathing problems during sleep may require noninvasive ventilation, which involves breathing support with a mask or similar device. Medical devices like a pacemaker or implantable cardioverter defibrillator (ICD) are advised for individuals with cardiological complications.
It covers the details of conventional and current medical therapies and diagnoses available in the myotonic dystrophy (DM) market to treat the condition. It also provides country-wise treatment guidelines and algorithms across the United States, Europe, and Japan.
The myotonic dystrophy (DM) epidemiology division provides insights into the historical and current patient pool and the forecasted trend for every 7MM. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of KOL. The report also provides the prevalent patient pool, trends, and assumptions.
The disease epidemiology covered in the report provides historical and forecasted myotonic dystrophy (DM) epidemiology segmented as the diagnosed prevalent cases of myotonic dystrophy (DM), type-specific diagnosed cases of myotonic dystrophy (DM), type-specific diagnosed cases of DM1, age-specific diagnosed cases of DM, and comorbidity associated diagnosed cases with myotonic dystrophy (DM). The report includes the prevalent myotonic dystrophy (DM) scenario in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2019 to 2032.
The epidemiology segment also provides the myotonic dystrophy (DM) epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
The total diagnosed prevalent patient population of myotonic dystrophy (DM) in the 7MM countries was close to 80,000 cases in 2021.
As per the estimates, the US had the highest patient population of myotonic dystrophy (DM) in 2021. Among the EU5 countries, Germany had the highest diagnosed patient population of myotonic dystrophy (DM), with over 9,000 cases, followed by France in 2021. On the other hand, Spain had the lowest diagnosed prevalent patient population of myotonic dystrophy (DM), with approximately 6,000 cases in 2021.
The drug chapter segment of the myotonic dystrophy (DM) report encloses the detailed analysis of myotonic dystrophy (DM) marketed drugs and late-stage (Phase III, Phase II/III, Phase II, and Phase I/II) pipeline drugs. It also helps understand the myotonic dystrophy (DM) clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.
The current standard of care lacks disease-modifying therapy. Management consists primarily of monitoring for complications and standard of care (assistive devices, hormone therapy, and pain medication). A few clinical studies have systematically evaluated the use of therapeutic agents in DM. The lack of scientific evidence paired with the multi-systematic and highly variable presentation of the disease makes identifying and selecting appropriate medications particularly challenging for prescribing physicians.
Thus, there is an urgent need to identify existing medications that are potentially safe and effective for DM patients and evaluate these therapies using appropriate clinical trials.
Drug developers are gradually shifting their attention toward myotonic dystrophy (DM) to meet the patient pool's current demands and counter the unmet needs of the therapeutic market.
Several companies are working robustly on many new therapies, such as AMO-02 (AMO Pharma Limited), mexiletine (Lupin Ltd.), and pitolisant (Harmony Biosciences, LLC).
AMO-02 (AMO Pharma Limited) is in development for treating congenital myotonic dystrophy (CDM) and has the potential for use in additional CNS, neuromuscular and other orphan indications. It has a dual mechanism disrupting the pathogenic RNA repeat in CDM1 and inhibiting excess levels of the kinase GSK3B. Currently, the company conducts two Phase II/III studies in children and adolescents with CDM.
The myotonic dystrophy (DM) market outlook of the report builds a detailed comprehension of the historical, current, and forecasted myotonic dystrophy (DM) market trends by analyzing the impact of current therapies on the market, unmet needs, and demand for better technology.
This segment gives a thorough detail of the myotonic dystrophy (DM) market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need for the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market, and KOL view. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.
As per DelveInsight, the myotonic dystrophy (DM) market in 7MM is expected to change in 2019-2032.
This section includes a glimpse of the myotonic dystrophy (DM) market in 7MM. In 2021, the myotonic dystrophy (DM) market size was approximately USD 80 million in the 7MM.
This section provides the total myotonic dystrophy (DM) market size. It also provides the market size of myotonic dystrophy (DM) by therapies in the United States.
The United States accounts for the highest market size of myotonic dystrophy (DM) than the EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.
This section provides the total myotonic dystrophy (DM) market size. It also provides myotonic Dystrophy (DM) market size by therapies in Germany, France, Italy, Spain, and the United Kingdom.
This section provides the total myotonic dystrophy (DM) market size. It also provides the market size of myotonic dystrophy (DM) by therapies in Japan.
This section focuses on the uptake rate of potential drugs launched or expected to be launched in the market during 2019-2032. The analysis covers myotonic dystrophy (DM) market uptake by drugs, patient uptake by therapies, and sales of each drug.
This helps in understanding the drugs with the most rapid uptake and the reasons behind the maximal use of new drugs and allows the comparison of the drugs based on market share and size, which again will be useful in investigating factors important in the market uptake and making financial and regulatory decisions.
The report provides insights into different therapeutic candidates in Phase III, Phase II/III, Phase II, and Phase I/II. It also analyses myotonic dystrophy (DM)'s key players in developing targeted therapeutics.
Major players include: AMO Pharma Limited, Lupin Ltd., Harmony Biosciences, and others whose key products are expected to get launched in the US market by 20XX.
The report covers collaborations, acquisitions, mergers, licensing, and patent details for emerging myotonic dystrophy (DM) therapies.
To keep up with current market trends, we take KOLs and SMEs' opinions working in the myotonic dystrophy (DM) domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps understand and validate current and emerging treatment patterns or myotonic dystrophy (DM) market trends. This will support the clients in potential novel treatments by identifying the overall scenario of the market and the unmet needs.
We perform a competitive and market intelligence analysis of the myotonic dystrophy (DM) market using various competitive intelligence tools: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.