The Myotonic dystrophy market report also offers comprehensive insights into the Myotonic dystrophy market size, share, Myotonic dystrophy epidemiology, emerging therapies, market drivers and barriers, ongoing clinical trials, key collaboration in the space, market uptake by key therapies and companies actively pushing Myotonic dystrophy market size growth forward.
Recent breakthroughs in the myotonic dystrophy market are:
December 2024 Updates:
-
PepGen Inc.: A study is underway to assess the effects of the investigational drug PGN-EDODM1, focusing on the safety and tolerability of multiple administrations of PGN-EDODM1 in individuals with myotonic dystrophy type 1 (DM1) compared to a placebo.
-
Avidity Biosciences Inc.: A clinical trial is being conducted with a 6-week screening period followed by a 54-week treatment phase, totaling approximately 60 weeks. Participants are randomized to receive intravenous infusions of either del-desiran or a placebo at 8-week intervals for a total of seven doses. The final dose is scheduled for Week 48, with the last assessment at Week 54.
-
Vertex Pharmaceuticals: A Phase 1/2 study is underway to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 in adults with DM1. The trial is randomized, double-blind, and placebo-controlled, involving both single- and multiple-dose escalations.
November 2024 Update:
-
ARTHEx Biotech S.L.: The FDA has granted Rare Pediatric Disease Designation (RPD) to ATX-01 for the treatment of myotonic dystrophy type 1 (DM1), a rare neuromuscular disorder. This designation follows the earlier Orphan Drug Designation (ODD) granted by U.S. and European regulatory authorities.
Some of the key highlights from the Myotonic dystrophy Market Insights Report:
-
Several key pharmaceutical companies, including Lupin, AMO Pharma Limited, Harmony Biosciences, LLC, and others, are developing novel products to improve the Myotonic dystrophy treatment outlook.
-
The Myotonic Dystrophy market is expected to experience steady growth throughout the forecast period (2024–2034). In the seven major markets (7MM), the Myotonic Dystrophy treatment market size is projected to expand, driven by improved diagnostic capabilities and the introduction of new therapies.
-
According to DelveInsight’s analysis, approximately 51% of the diagnosed prevalent Myotonic Dystrophy cases in the 7MM are in the United States. In 2023, the U.S. accounted for an estimated 54,068 diagnosed cases of Myotonic Dystrophy. The total treatment market is anticipated to grow further during the forecast period, supported by the development and potential approval of emerging therapies such as Tideglusib (AMO-02).
-
The Myotonic Dystrophy therapeutics market is forecasted to grow at a significant compound annual growth rate (CAGR) of 18.4% from 2020 to 2034. This substantial growth reflects increasing demand for advanced treatment options and highlights rising investments in addressing this challenging condition.
-
Several factors contribute to this growth, including the rising prevalence of genetic disorders, increased awareness of rare diseases, advancements in genomic research, and growing investment in rare disease therapies. However, challenges such as limited research funding, high costs associated with innovative treatments, and stringent regulatory requirements pose significant barriers. Additionally, the unmet need for effective Myotonic Dystrophy treatments underscores a critical gap in the current therapeutic landscape, presenting opportunities for innovation to meet the needs of a diverse patient population.
-
As per DelveInsight analysis, the Myotonic dystrophy market is anticipated to witness growth at a considerable CAGR
Strategise your business goals by understanding market dynamics @ Myotonic dystrophy Market Landscape
Myotonic dystrophy Overview
Myotonic dystrophy types 1 and 2 are among the most prevalent forms of muscular dystrophy that typically present during adulthood. Recognizing the clinical distinctions between these two types is critical for selecting the most suitable treatment approaches for patients. While no disease-modifying therapies currently exist, various symptomatic treatments are available to help manage the condition. Encouragingly, advancements in next-generation therapies may soon offer new possibilities for individuals affected by the disease. Effective symptom management is vital, as it can greatly alleviate patient suffering and improve overall quality of life. Additionally, regular disease monitoring is essential to detect and address potential complications during critical periods.
Myotonic dystrophy is a type of muscular dystrophy, a group of disorders marked by the weakening and degeneration of specific voluntary muscles in the body. Each form of muscular dystrophy has unique characteristics, including variations in muscle fiber size, muscle fiber necrosis, scar tissue formation, and inflammation, as observed in muscle biopsies from affected patients.
There are two main types of myotonic dystrophy: type 1 (commonly known as Steinert disease, after Dr. Steinert who first described it in 1909) and type 2 (referred to as Ricker syndrome or proximal myotonic dystrophy, PROMM). Myotonic dystrophy should be suspected in patients presenting with muscle weakness, a family history of the condition, and characteristic physical exam findings.
The gold standard for diagnosing myotonic dystrophy is genetic testing for CTG repeat expansions. Additional diagnostic tests may include serum creatine kinase levels, hepatobiliary function tests, muscle biopsies, and electrocardiograms to evaluate potential cardiomyopathy. These tests are often performed before genetic confirmation to assess the overall impact of the disease on the patient’s health.
Do you know the treatment paradigms for different countries? Download our Myotonic dystrophy Market Sample Report
Myotonic dystrophy Epidemiology Insights
-
Within the 7MM, the EU4 and the UK collectively accounted for approximately 353,191 diagnosed prevalent cases of myotonic dystrophy, with this number projected to grow during the forecast period (2024–2034).
-
Among the EU4 and the UK, Germany had the highest diagnosed prevalence of myotonic dystrophy in 2023, with 8,938 cases, followed by the UK and France. Conversely, Spain had the lowest diagnosed prevalence within this group.
-
In Japan, there were approximately 12,735 diagnosed prevalent cases of myotonic dystrophy in 2023, and this number is anticipated to grow at a notable CAGR.
Myotonic dystrophy Epidemiology Segmentation
DelveInsight’s Myotonic dystrophy market report is prepared on the basis of epidemiology model. It offers comprehensive insights to the Myotonic dystrophy historical patient pools and forecasted Myotonic dystrophy patients. The report provides in-depth data of various subtypes and for the same epidemiology is segmented further. The Myotonic dystrophy Market report proffers epidemiological analysis for the study period 2020-34 in the 7MM segmented into:
-
Myotonic dystrophy Prevalence
-
Age-Specific Myotonic dystrophy Prevalence
-
Gender-Specific Myotonic dystrophy Prevalence
-
Diagnosed and Treatable Cases of Myotonic dystrophy
Visit for more @ Myotonic dystrophy Epidemiological Insights
Myotonic dystrophy Market Outlook
Myotonic dystrophy is a dominantly inherited form of muscular dystrophy that impacts both muscles and various body systems. It can cause symptoms such as early cataracts, myotonia, muscle weakness and atrophy, fatigue, excessive daytime sleepiness, central or obstructive apnea, respiratory failure, cardiac arrhythmias, insulin resistance, dysphagia, mood disorders, and more.
There are two types of myotonic dystrophy:
– Type 1 (DM1): Caused by the expansion of a CTG triplet repeat in the DMPK gene.
– Type 2 (DM2): Caused by the expansion of a CCTG tetramer repeat in the CNBP gene.
The symptoms of DM2 are generally milder than those of DM1 and lack a congenital form. Clinical manifestations of DM2 typically begin after the third decade of life, presenting with muscle-related symptoms such as weakness, musculoskeletal pain, stiffness, myotonia, fatigue, and exercise intolerance, which are often the reasons for initial neurological referrals. It is essential to adopt tailored interventions to improve patients’ quality of life (QoL).
Currently, no therapies are approved to cure or slow the progression of myotonic dystrophy. However, symptomatic treatments are available to alleviate suffering and enhance QoL.
Symptom Management:
– Myotonia: The most commonly treated symptom, managed with off-label antimyotonic agents such as mexiletine, lamotrigine, carbamazepine, oxcarbazepine, flecainide, propafenone, phenytoin, and ranolazine.
– Chronic Muscle Pain: Managed using the WHO four-step ladder for pain management, beginning with NSAIDs. Additional therapies may include anticonvulsants (pregabalin, gabapentin), antidepressants (duloxetine, amitriptyline, nortriptyline), muscle relaxants (baclofen, tizanidine), or topical agents (lidocaine or capsaicin patches).
– Excessive Daytime Sleepiness: More prevalent in DM1 and managed with medications like methylphenidate and modafinil, which are well-tolerated and effective.
– Diabetic Symptoms: Managed with anti-diabetic drugs like metformin to normalize blood sugar levels.
Additionally, various medications, rehabilitative therapies, surgeries, and medical devices are used to address symptoms and complications.
Emerging Therapies:
Mexiletine, currently used off-label, is under development for formal approval and is in the pipeline for myotonic dystrophy treatment. This underscores its potential efficacy and highlights the need for its recognition as an authorized therapy in regions such as the US, the EU, and Japan.
Myotonic dystrophy Emerging Drugs
-
Tideglusib (AMO-02): AMO Pharma
-
Delpacibart etedesiran (AOC-1001): Avidity Biosciences, Inc.
Myotonic dystrophy Key Companies
-
Lupin, AMO Pharma Limited, Harmony Biosciences, LLC, and others
For more information, visit Myotonic dystrophy Market Analysis, Patient Pool, and Emerging Therapies
Scope of the Myotonic dystrophy Market Report:
-
11 Years Forecast
-
7MM Coverage
-
Descriptive overview of Myotonic dystrophy, causes, signs and symptoms, diagnosis, treatment
-
Comprehensive insight into Myotonic dystrophy epidemiology in the 7MM
-
Myotonic dystrophy marketed and emerging therapies
-
Myotonic dystrophy companies
-
Myotonic dystrophy market drivers and barriers
Table of Contents:
1 Myotonic dystrophy Market Key Comprehensive Insights
2 Myotonic dystrophy Market Report Introduction
3 Competitive Intelligence Analysis for Myotonic dystrophy
4 Myotonic dystrophy Market Analysis Overview at a Glance
5 Executive Summary of Myotonic dystrophy
6 Myotonic dystrophy Epidemiology and Market Methodology
7 Myotonic dystrophy Epidemiology and Patient Population
8 Myotonic dystrophy Patient Journey
9 Myotonic dystrophy Treatment Algorithm, Myotonic dystrophy Current Treatment, and Medical Practices
10 Key Endpoints in Myotonic dystrophy Clinical Trials
11 Myotonic dystrophy Marketed Therapies
12 Myotonic dystrophy Emerging Therapies
13 Myotonic dystrophy: 7 Major Market Analysis
14 Attribute analysis
15 Access and Reimbursement Overview of Myotonic dystrophy
16 Myotonic dystrophy Market Key Opinion Leaders Reviews
18 Myotonic dystrophy Market Drivers
19 Myotonic dystrophy Market Barriers
20 SWOT Analysis
21 Disclaimer
22 DelveInsight Capabilities
23 About DelveInsight
Related Reports:
Myotonic dystrophy Epidemiology 2034
DelveInsight’s “Myotonic dystrophy – Epidemiology Forecast to 2034” report delivers an in-depth understanding of the disease, historical and forecasted Myotonic dystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
Myotonic dystrophy Pipeline 2024
“Myotonic dystrophy Pipeline Insights, 2024” report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Myotonic dystrophy market. A detailed picture of the Myotonic dystrophy pipeline landscape is provided, which includes the disease overview and Myotonic dystrophy treatment guidelines.
Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Kritika Rehani
Email: Send Email
Phone: +14699457679
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: Nevada
Country: United States
Website: https://www.delveinsight.com/