Myotonic Dystrophy - Epidemiology Forecast - 2032

Myotonic Dystrophy - Epidemiology Forecast - 2032

DelveInsight’s ‘Myotonic Dystrophy (DM) – Epidemiology Forecast – 2032’ report delivers an in-depth understanding of the historical and forecasted epidemiology of myotonic dystrophy in the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

Myotonic Dystrophy: Disease Understanding

Myotonic Dystrophy Overview

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.

People with DM often experience prolonged muscle tensing (myotonia) and cannot relax certain muscles after use: For example, someone with DM may have difficulty letting go of someone’s hand after shaking it. Disease severity may vary greatly according to DM type and the age of the person with the disorder, even among members of the same family. However, in general, symptoms progress slowly. DM is the most common form of muscular dystrophy that begins in adulthood, usually in the 20s or 30s. In some cases, babies are born with a variation of DM1 called congenital myotonic dystrophy (CDM).

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 the 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. Treatment may require the coordinated efforts of a team of specialists. Pediatricians, orthopedists, neurologists, and specialists who assess and treat cardiologists, pulmonologists, ophthalmologists, speech pathologists, and other healthcare professionals may need to systematically and comprehensively plan an affected individual’s treatment.

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. Some infants with CDM require a gastronomy tube, a thin tube inserted directly into the stomach through a small surgical opening to ensure that infants receive the required nutrients but avoid the risk of aspiration.

Additionally, nonpharmacological strategies like physical and occupational therapy, psychosocial support, and genetic counseling for affected individuals and their families can be of benefit. Supportive aids such as braces, walkers, or wheelchairs may help those who experience muscle weakness or fatigue problems. Research is being conducted in several geographies to create new therapeutic options for the permanent cure of DM.

Epidemiology

The myotonic dystrophy epidemiology division provides insights into the historical and current patient pool and the forecast trend for every seven major countries. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the report also provides the diagnosed patient pool and their trends, along with assumptions undertaken.

Key Findings

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 myotonic dystrophy (DM), and comorbidity associated diagnosed cases with myotonic dystrophy (DM). The report includes the prevalent scenario of myotonic dystrophy in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2019 to 2032.

Country-wise Myotonic Dystrophy Epidemiology

The epidemiology segment also provides the myotonic dystrophy 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 prevalent 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.

Scope of the Report

Myotonic dystrophy report covers a detailed overview explaining its causes, symptoms and classification, pathophysiology, diagnosis, and treatment patterns.

Myotonic dystrophy epidemiology report and model provide an overview of the risk factors and global trends of myotonic dystrophy in the seven major markets (7MM: The US, France, Germany, Italy, Spain, UK, and Japan).

The report provides insight into the historical and forecasted patient pool of myotonic dystrophy in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan.

The report helps recognize the growth opportunities in the 7MM concerning the patient population.

The report assesses the disease risk and burden and highlights the unmet needs of myotonic dystrophy.

The report describes myotonic dystrophy epidemiology by prevalent cases of myotonic dystrophy in the 7MM.

The report describes the epidemiology of myotonic dystrophy by diagnosed cases of myotonic dystrophy in the 7MM.

The report provides the segmentation of the myotonic dystrophy epidemiology by the prevalence of myotonic dystrophy (DM) based on symptoms in the 7MM.

The report provides the segmentation of the myotonic dystrophy epidemiology by the genotype of myotonic dystrophy (DM) diagnosed patients in the 7MM.

The report segments the myotonic dystrophy epidemiology by comorbidity associated with myotonic dystrophy (DM) of myotonic dystrophy in the 7MM.

Report Highlights

11-year Forecast of Myotonic Dystrophy Epidemiology

7MM Coverage

Diagnosed Prevalent cases of Myotonic Dystrophy

Type-specific Diagnosed cases of Myotonic Dystrophy

Type-specific Diagnosed cases of Myotonic Dystrophy Type 1

Age-specific Diagnosed cases of Myotonic Dystrophy

Comorbidity associated with Diagnosed cases with Myotonic Dystrophy

KOL Views

We interview KOLs and obtain SMEs’ opinions through primary research to fill the data gaps and validate our secondary research. The opinion helps understand the total patient population and current treatment pattern. This will support the clients in potential novel treatments by identifying the overall scenario of the indications.

Key Questions Answered

What are the major factors that will drive the change in the patient population in myotonic dystrophy during the forecast period (2019–2032)?

What key findings about myotonic dystrophy epidemiology across 7MM, and which country will have the highest number of patients during the forecast period (2019–2032)?

What would be the total number of patients with myotonic dystrophy across the 7MM forecast period (2019–2032)?

Among the EU5 countries, which country will have the highest number of patients during the forecast period (2019–2032)?

At what CAGR is the patient population expected to grow in the 7MM forecast period (2019–2032)?

What are the disease risk, burdens, and unmet needs of myotonic dystrophy?

What are the currently available treatments for myotonic dystrophy?

Reasons to buy

Myotonic dystrophy epidemiology report will allow the user to:

Develop business strategies by understanding the trends shaping and driving the global myotonic dystrophy market.

Quantify patient populations in the global myotonic dystrophy market to improve product design, pricing, and launch plans.

Understand the magnitude of the myotonic dystrophy population by its diagnosed prevalent cases.

Understand the magnitude of the myotonic dystrophy population by its type-specific diagnosed cases.

Understand the magnitude of the myotonic dystrophy population by its type-specific diagnosed cases of myotonic dystrophy type 1.

Understand the magnitude of the myotonic dystrophy population by its age-specific diagnosed cases of myotonic dystrophy.

Understand the magnitude of the myotonic dystrophy population by its comorbidity associated diagnosed cases with myotonic dystrophy.

The myotonic dystrophy epidemiology report and model were written and developed by Masters and PhD level epidemiologists.

The myotonic dystrophy epidemiology model developed by DelveInsight is easy to navigate, interactive with dashboards, and epidemiology based on transparent and consistent methodologies. Moreover, the model supports the data presented in the report and showcases disease trends over an 11-year forecast period using reputable sources.

Key Assessments

Patient Segmentation

Disease Risk and Burden

Risk of disease by the segmentation

Factors driving growth in a specific patient population

Geographies Covered

The United States

EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Japan