Myotonic Dystrophy Pipeline, Clinical Trials Assessment, and FDA Approvals 2023 (Updated)

Myotonic Dystrophy Pipeline, Clinical Trials Assessment, and FDA Approvals 2023 (Updated)

DelveInsight’s, “Myotonic Dystrophy Pipeline Insight, 2023,” report provides comprehensive insights about 20+ companies and 22+ pipeline drugs in the Myotonic Dystrophy pipeline landscape. It covers the Myotonic Dystrophy pipeline drug profiles, including clinical 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.

 

Key Takeaways from the Myotonic Dystrophy Pipeline Report

  • DelveInsight’s Myotonic Dystrophy pipeline report depicts a robust space with 20+ active players working to develop 22+ pipeline therapies for Myotonic Dystrophy treatment.
  • The leading companies working in the Myotonic Dystrophy Market include AMO Pharma, Harmony Biosciences, Dyne Therapeutics, Avidity Biosciences, Enzerna, Juvena Therapeutics, NeuBase Therapeutics, Ionis Pharmaceuticals, Expansion Therapeutics Inc., Insmed Incorporated, Lupin Ltd, Gilead Sciences, Sanguine Biosciences, and others.
  • Promising Myotonic Dystrophy Pipeline Therapies in the various stages of development include Tideglusib, IONIS-DMPKRx, ERX-963, DYNE-101, rhIGF-I/rhIGFBP-3, Pitolisant Oral Tablet, and others.
  • June 2023: Dyne Therapeutics announced a study of phase 1 & 2 clinical trials for DYNE-101. The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1).
  • September 2023: AMO Pharma Limited announced a study of phase 2 clinical trials for Tideglusib. This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).

 

Request a sample and discover the recent advances in Myotonic Dystrophy Treatment Drugs @ Myotonic Dystrophy Pipeline Report

 

In the Myotonic Dystrophy pipeline report, detailed description of the drug is given which includes mechanism of action of the drug, Myotonic Dystrophy clinical trials studies, Myotonic Dystrophy NDA approvals (if any), and product development activities comprising the technology, collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

 

Myotonic Dystrophy Overview

Myotonic dystrophy (DM) is considered a subgroup of myopathy and the most common type of muscular dystrophy that begins in adulthood. There are two major forms recognized based on clinical and molecular presentation: Myotonic dystrophy type I (DM1), known as Steinert disease, and myotonic dystrophy type II (DM2), or proximal myotonic myopathy which is a milder variety of DMI.

 

Find out more about Myotonic Dystrophy Therapeutics Assessment @ Myotonic Dystrophy Preclinical and Discovery Stage Products

 

Myotonic Dystrophy Emerging Drugs Profile

  • Tideglusib: AMO Pharma
  • Pitolisant: Harmony Biosciences
  • DYNE-101: Dyne Therapeutics

 

Myotonic Dystrophy Pipeline Therapeutics Assessment

There are approx. 20+ key companies which are developing the Myotonic Dystrophy therapies. The Myotonic Dystrophy companies which have their Myotonic Dystrophy drug candidates in the most advanced stage, i.e. phase II/III include, AMO Pharma.

 

Learn more about the emerging Myotonic Dystrophy Pipeline Therapies @ Myotonic Dystrophy Clinical Trials Assessment

 

Scope of the Myotonic Dystrophy Pipeline Report

  • Coverage- Global
  • 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
  • Myotonic Dystrophy Companies- AMO Pharma, Harmony Biosciences, Dyne Therapeutics, Avidity Biosciences, Enzerna, Juvena Therapeutics, NeuBase Therapeutics, Ionis Pharmaceuticals, Expansion Therapeutics Inc., Insmed Incorporated, Lupin Ltd, Gilead Sciences, Sanguine Biosciences, and others.
  • Myotonic Dystrophy Pipeline Therapies- Tideglusib, IONIS-DMPKRx, ERX-963, DYNE-101, rhIGF-I/rhIGFBP-3, Pitolisant Oral Tablet, and others.

 

Dive deep into rich insights for new drugs for Myotonic Dystrophy treatment, Visit @ Myotonic Dystrophy Market Drivers and Barriers, and Future Perspective

 

Table of Content

  1. Introduction
  2. Executive Summary
  3. Myotonic Dystrophy: Overview
  4. Pipeline Therapeutics
  5. Therapeutic Assessment
  6. Myotonic Dystrophy– DelveInsight’s Analytical Perspective
  7. Mid Stage Products (Phase II/III)
  8. Tideglusib: AMO Pharma
  9. Drug profiles in the detailed report…..
  10. Mid Stage Products (Phase II)
  11. Pitolisant: Harmony Biosciences
  12. Drug profiles in the detailed report…..
  13. Early Stage Products (Phase I/II)
  14. DYNE-101: Dyne Therapeutics
  15. Drug profiles in the detailed report…..
  16. Preclinical and Discovery Stage Products
  17. Drug Name: Company Name
  18. Drug profiles in the detailed report…..
  19. Inactive Products
  20. Myotonic Dystrophy Key Companies
  21. Myotonic Dystrophy Key Products
  22. Myotonic Dystrophy- Unmet Needs
  23. Myotonic Dystrophy- Market Drivers and Barriers
  24. Myotonic Dystrophy- Future Perspectives and Conclusion
  25. Myotonic Dystrophy Analyst Views
  26. Myotonic Dystrophy Key Companies
  27. Appendix

 

For further information on the Myotonic Dystrophy pipeline therapeutics, reach out to Myotonic Dystrophy Unmet Needs and Analyst Views

 

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