Press release
Myotonic Dystrophy Clinical Trials and Studies 2025: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies by DelveInsight
Stay ahead with the latest insights! Download DelveInsight's comprehensive Myotonic Dystrophy Pipeline Report to explore emerging therapies, key Myotonic Dystrophy Companies, and future Myotonic Dystrophy treatment landscapes @ Myotonic Dystrophy Pipeline Outlook Report [https://www.delveinsight.com/sample-request/myotonic-dystrophy-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]
Key Takeaways from the Myotonic Dystrophy Pipeline Report
* In February 2025:- Avidity Biosciences Inc.:- A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1. The study consists of a Screening Period of up to 6 weeks and 54-week Treatment Period. The anticipated duration is approximately 60 weeks.
* In January 2025:- ARTHEx Biotech S.L.:- The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated. The trial will compare the safety and tolerability of ATX-01 and a matching placebo.
* DelveInsight's Myotonic Dystrophy pipeline report depicts a robust space with 20+ active players working to develop 27+ pipeline therapies for Myotonic Dystrophy treatment.
* The leading Myotonic Dystrophy Companies such as AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Aparito , and others.
* Dystrophy Therapies such as AOC 1001 (del-desiran), Tideglusib, ATX-01, IONIS-DMPKRx, VX-670, DYNE-101 , and others.
Discover how the Myotonic Dystrophy treatment paradigm is evolving. Access DelveInsight's in-depth Myotonic Dystrophy Pipeline Analysis for a closer look at promising breakthroughs @ Myotonic Dystrophy Clinical Trials and Studies [https://www.delveinsight.com/sample-request/myotonic-dystrophy-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]
Myotonic Dystrophy Emerging Drugs Profile
* Tideglusib: AMO Pharma
AMO-02 (tideglusib) is in development for the treatment of congenital myotonic dystrophy and has potential for use in additional CNS, neuromuscular and other orphan indications. AM0-02 is a clinical stage investigational medicine for the treatment of the severe form of congenital myotonic dystrophy known as DM1 or Steinert disease. In cellular and animal models of DM1 as well as in muscle biopsies from patients, activity of glycogen synthase kinase 3 beta (GSK3Ss) has been shown to increase.
* Pitolisant: Harmony Biosciences
Pitolisant (WAKIX) is a selective histamine 3 (H3) receptor antagonist/inverse agonist. The mechanism of action of WAKIX is unclear; however, its efficacy could be mediated through its activity at H3 receptors, thereby increasing the synthesis and release of histamine, a wake promoting neurotransmitter. WAKIX was designed and developed by Bioprojet (France). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States. Currently the drug is being investigated in Phase II stage of Clinical trial evaluation for the treatment of Myotonic Dystrophy.
* ARO-DM1: Arrowhead Pharmaceuticals, Inc.
ARO-DM1 is an RNA interference (RNAi) conjugate designed to specifically silence DMPK mRNA in skeletal muscle. Published literature suggests that the silencing of aberrantly transcribed DMPK mRNA using ARO-DM1 may halt CUGexp-related spliceopathies in patients with DM1 leading to improved muscle strength and function. It belongs to a class of medicines called RNA therapeutics. The drug candidate is administered intravenously in the body. ARO-DM1 is currently in Phase I stage of development for the treatment of myotonic dystrophy.
Get a detailed analysis of the latest innovations in the Myotonic Dystrophy pipeline. Explore DelveInsight's expert-driven report today! @ Myotonic Dystrophy Unmet Needs [https://www.delveinsight.com/sample-request/myotonic-dystrophy-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]
Myotonic Dystrophy Companies
AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Aparito, and others.
Myotonic Dystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
* Oral
* Intravenous
* Subcutaneous
* Parenteral
* Topical
Myotonic Dystrophy Products have been categorized under various Molecule types such as
* Recombinant fusion proteins
* Small molecule
* Monoclonal antibody
* Peptide
* Polymer
* Gene therapy
Download DelveInsight's latest report to gain strategic insights into upcoming Myotonic Dystrophy Therapies and key Myotonic Dystrophy Developments @ Myotonic Dystrophy Market Drivers and Barriers, and Future Perspectives [https://www.delveinsight.com/sample-request/myotonic-dystrophy-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]
Scope of the Myotonic Dystrophy Pipeline Report
* Coverage- Global
* Myotonic Dystrophy Companies- AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Aparito , and others.
* Myotonic Dystrophy Therapies- AOC 1001 (del-desiran), Tideglusib, ATX-01, IONIS-DMPKRx, VX-670, DYNE-101 , and others.
* Myotonic Dystrophy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
* Myotonic Dystrophy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
Which companies are leading the race in Myotonic Dystrophy drug development? Find out in DelveInsight's exclusive Myotonic Dystrophy Pipeline Report-access it now! @ Myotonic Dystrophy Emerging Drugs and Major Companies [https://www.delveinsight.com/sample-request/myotonic-dystrophy-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]
Table of Content
* Introduction
* Executive Summary
* Myotonic Dystrophy: Overview
* Pipeline Therapeutics
* Therapeutic Assessment
* Myotonic Dystrophy- DelveInsight's Analytical Perspective
* Late Stage Products (Phase II/III)
* Tideglusib: AMO Pharma
* Drug profiles in the detailed report.....
* Mid Stage Products (Phase II)
* Pitolisant: Harmony Biosciences
* Drug profiles in the detailed report.....
* Early Stage Products (Phase I)
* Comparative Analysis
* ARO-DM1: Arrowhead Pharmaceuticals, Inc.
* Drug profiles in the detailed report.....
* Preclinical and Discovery Stage Products
* Drug name: Company name
* Drug profiles in the detailed report.....
* Inactive Products
* Myotonic Dystrophy Key Companies
* Myotonic Dystrophy Key Products
* Myotonic Dystrophy- Unmet Needs
* Myotonic Dystrophy- Market Drivers and Barriers
* Myotonic Dystrophy- Future Perspectives and Conclusion
* Myotonic Dystrophy Analyst Views
* Myotonic Dystrophy Key Companies
* Appendix
About Us
DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.
Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Yash Bhardwaj
Email:Send Email [https://www.abnewswire.com/email_contact_us.php?pr=myotonic-dystrophy-clinical-trials-and-studies-2025-ema-pdma-fda-approvals-mechanism-of-action-roa-nda-ind-and-companies-by-delveinsight]
Phone: 9650213330
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: Nevada
Country: United States
Website: https://www.delveinsight.com/report-store/vitreoretinal-surgery-devices-market
Legal Disclaimer: _Information contained on this page is provided by an independent third-party content provider. ABNewswire makes no warranties or responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you are affiliated with this article or have any complaints or copyright issues related to this article and would like it to be removed, please contact retract@swscontact.com_
This release was published on openPR.
Permanent link to this press release:
Copy
Please set a link in the press area of your homepage to this press release on openPR. openPR disclaims liability for any content contained in this release.
You can edit or delete your press release Myotonic Dystrophy Clinical Trials and Studies 2025: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies by DelveInsight here
News-ID: 3859976 • Views: …
More Releases from ABNewswire
A Little Laughter, A Lot of Heart: Hannah's Docs & Ducks Reminds Families Why Re …
There's something special about a book that makes both kids and adults laugh at the same time. Docs & Ducks: A Quakery of Poems by Hannah is exactly that kind of book. It's light, funny, and a little bit thoughtful in ways you don't expect until you've turned the page.
The poems are short and easy to read, the kind you can slip into a bedtime routine or a classroom break.…
Matthew Mettias - Hawaii-born Sound Artist, Poet, and Creative Technologist - Em …
STANFORD, CA - November 18, 2025 - Hawaii-born, California-based sound artist, poet, composer, producer, and creative technologist, Matthew Mettias, is rapidly gaining international attention with a creative practice that blends island memory, cinematic sound, and ecopsychic healing. This month, his newest release reached #8 on Iceland's Top 100 Songs, marking a global breakthrough for the rising artist.
Raised in Honolulu and now a Stanford University alumnus, Mettias bridges his roots with…
XMO Corp CTO & Cofounder Brian Alvara to Speak on Digital Literacy and Tech Flue …
Washington, D.C. - November 18, 2025 - XMO Corp proudly announces that Brian Alvara, Chief Technology Officer and Cofounder, will be a featured speaker at the upcoming panel discussion, hosted by 2025 CHCI Tech Summit "?Hablas Tech?/Do You Speak Tech?", taking place on November 19, 2025, from 10:45 AM - 11:45 AM ET at Capital Turnaround, 700 M St SE, Washington, DC 20003.
The session will address one of the most…
Atlanta Hawks To Honor Bob Rathbun For 30 Seasons Of Calling Hawks Basketball
Team To Celebrate FanDuel Sports Network Southeast Play-by-Play Voice and Georgia Sports Hall-of-Famer
Image: https://www.abnewswire.com/upload/2025/11/409c3f8604a74e2eec269ecc8b06e22a.jpg
ATLANTA - The Atlanta Hawks will honor Emmy-Award winning television play-by-play voice Bob Rathbun, who is celebrating his 30th season with the team, tonight during the team's contest against the Detroit Pistons at State Farm Arena. Since joining the organization in 1996, Rathbun has become an iconic figure in Atlanta sports, establishing an enduring bond with fans…
More Releases for Dystrophy
Cone Rod Dystrophy Market Innovations & Forecast to 2034
The Cone Rod Dystrophy Market is gaining notable momentum as advancements in genetic testing, stem cell research, and retinal gene therapy open new possibilities for treating this rare inherited eye disorder. With rising global awareness, increased funding for retinal disease programs, and expanding diagnostic capabilities, the market is poised for significant growth through 2034. Below is a complete market overview including key players, segmentation, clinical progress, and major growth drivers.
Download…
Duchenne Muscular Dystrophy Pipeline Outlook Report 2024
DelveInsight's, "Duchenne Muscular Dystrophy Pipeline Insight 2024" report provides comprehensive insights about 75+ companies and 75+ pipeline drugs in the Duchenne Muscular Dystrophy pipeline landscape. It covers the Duchenne Muscular Dystrophy pipeline drug profiles, including Duchenne Muscular Dystrophy clinical trials and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
…
Duchenne Muscular Dystrophy Treatmcent Market
Global Duchenne Muscular Dystrophy Treatmcent Market Set for Robust Growth During Forecast Period
The global Duchenne Muscular Dystrophy Treatment Market is poised to witness significant growth at a high Compound Annual Growth Rate (CAGR) during the forecast period of 2023 to 2030. Duchenne muscular dystrophy (DMD) stands as a genetic disorder marked by progressive muscle degeneration and weakness, with therapeutics aimed at addressing the absence of dystrophin, a crucial protein in…
Becker Muscular Dystrophy Market Research Report 2032
DelveInsight's "Becker Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of the Becker Muscular Dystrophy (BMD), historical and forecasted epidemiology as well as the Becker Muscular Dystrophy (BMD) market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
Becker Muscular Dystrophy (BMD) is an inherited muscle structure disorder that results in progressive deterioration of limbs, cardiac and skeletal muscles. However,…
Duchenne Muscular Dystrophy (DMD) Drugs Market Report
As per the research conducted by GME, the Duchenne Muscular Dystrophy (DMD) Drugs Market will grow with a CAGR value of 41.3 percent by 2026. The global market for Duchenne muscular dystrophy is foreseen to grow due to accelerated research and development activities, expanded patient awareness for effective treatments, and the launch of new medications Moreover, the disease's increasing prominence is likely to drive the global duchenne muscular dystrophy market…
Duchenne Muscular Dystrophy - Pipeline Review, H1 2017
ReportsWorldwide has announced the addition of a new report title Duchenne Muscular Dystrophy - Pipeline Review, H1 2017 to its growing collection of premium market research reports.
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Duchenne Muscular Dystrophy - Pipeline Review, H1 2017, provides an overview of the Duchenne Muscular Dystrophy (Genetic Disorders) pipeline landscape.
Duchenne muscular dystrophy is a condition which causes muscle weakness. DMD is an X-linked disorder.…
