LAS VEGAS, Nov. 27, 2024
An increase in research and development (R&D) activities in myotonic dystrophy is expected to drive market growth by enhancing the understanding of the disease, accelerating drug discovery, and creating innovative treatments. With more R&D, new therapeutic options such as gene therapies, antisense oligonucleotides, and targeted small molecules can be developed, addressing the unmet needs in myotonic dystrophy care. Increased R&D also attracts funding and partnerships, fostering a competitive environment that can lead to improved patient outcomes and market expansion.
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LAS VEGAS, Nov. 27, 2024 /PRNewswire/ -- DelveInsight's 'Myotonic Dystrophy Pipeline Insight 2024' report provides comprehensive global coverage of pipeline myotonic dystrophy therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the myotonic dystrophy pipeline domain.
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Key Takeaways from the Myotonic Dystrophy Pipeline Report
Request a sample and discover the recent advances in myotonic dystrophy treatment drugs @ Myotonic Dystrophy Pipeline Report
The myotonic dystrophy pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage myotonic dystrophy drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the myotonic dystrophy clinical trial landscape.
Myotonic Dystrophy Overview
Myotonic dystrophy is a genetic disorder characterized by progressive muscle wasting and weakness. It is caused by mutations in specific genes that lead to an abnormal expansion of repetitive DNA sequences. The two main types of myotonic dystrophy are Myotonic Dystrophy Type 1 (DM1) and Type 2 (DM2). DM1 is caused by a CTG repeat expansion in the DMPK gene, while DM2 is linked to a CCTG repeat expansion in the CNBP gene.
Symptoms of myotonic dystrophy can vary but often include muscle stiffness (myotonia), weakness, and atrophy, primarily affecting the distal muscles and those of the face. Additional symptoms may involve cataracts, cardiac arrhythmias, endocrine issues, and cognitive difficulties. Diagnosis typically involves a combination of clinical evaluation, family history assessment, and genetic testing to identify the specific mutations. Electromyography can also be used to detect myotonia.
Currently, there is no cure for myotonic dystrophy, and treatment focuses on managing symptoms. This may include physical therapy to maintain muscle function, medications to alleviate myotonia, and regular monitoring for cardiac issues. Genetic counseling is recommended for affected individuals and their families to understand the inheritance patterns and potential risks.
Find out more about myotonic dystrophy treatment drugs @ Drugs for Myotonic Dystrophy Treatment
A snapshot of the Myotonic Dystrophy Pipeline Drugs mentioned in the report:
Drugs
Company
Phase
MoA
RoA
AOC 1001
Avidity Biosciences
Phase III
Myotonin protein kinase expression inhibitors; RNA interference
Intravenous
Tideglusib
AMO Pharma
Phase III
Glycogen synthase kinase 3 beta inhibitors
Oral
Pitolisant
Harmony Biosciences
Phase II
Histamine H3 receptor antagonists; Histamine H3 receptor inverse agonists
Oral
PGN EDODM1
Pepgen Corporation
Phase II
RNA splicing modulators
Intravenous
ATX-01
Arthex Biotech
Phase I/II
MicroRNA inhibitor
Intravenous
DYNE-101
Dyne Therapeutics
Phase I/II
Myotonin protein kinase expression inhibitor
Intravenous
VX-670
Vertex Pharmaceuticals
Phase I/II
RNA inhibitors
Intravenous
ARO-DM1
Arrowhead Pharmaceuticals, Inc.
Phase I
Myotonin protein kinase expression inhibitors
Intravenous
Learn more about the emerging myotonic dystrophy pipeline therapies @ Myotonic Dystrophy Clinical Trials
Myotonic Dystrophy Therapeutics Assessment
The myotonic dystrophy pipeline report proffers an integral view of the myotonic dystrophy emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration.
Scope of the Myotonic Dystrophy Pipeline Report
Dive deep into rich insights for new drugs for myotonic dystrophy treatment, visit @ Myotonic Dystrophy Drugs
Table of Contents
1.
Myotonic Dystrophy Pipeline Report Introduction
2.
Myotonic Dystrophy Pipeline Report Executive Summary
3.
Myotonic Dystrophy Pipeline: Overview
4.
Analytical Perspective In-depth Commercial Assessment
5.
Myotonic Dystrophy Clinical Trial Therapeutics
6.
Myotonic Dystrophy Pipeline: Late-Stage Products (Pre-registration)
7.
Myotonic Dystrophy Pipeline: Late-Stage Products (Phase III)
8.
Myotonic Dystrophy Pipeline: Mid-Stage Products (Phase II)
9.
Myotonic Dystrophy Pipeline: Early-Stage Products (Phase I)
10.
Myotonic Dystrophy Pipeline Therapeutics Assessment
11.
Inactive Products in the Myotonic Dystrophy Pipeline
12.
Company-University Collaborations (Licensing/Partnering) Analysis
13.
Key Companies
14.
Key Products in the Myotonic Dystrophy Pipeline
15.
Unmet Needs
16.
Market Drivers and Barriers
17.
Future Perspectives and Conclusion
18.
Analyst Views
19.
Appendix
For further information on the myotonic dystrophy pipeline therapeutics, reach out @ Myotonic Dystrophy Treatment Drugs
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