(Albany, United States) As per DelveInsight’s assessment, globally, the Myotonic Dystrophy pipeline constitutes 20+ key companies continuously working towards developing 22+ Myotonic Dystrophy treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analysis DelveInsight.
In the Myotonic Dystrophy Pipeline Report, a 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.
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Key Takeaways from the Myotonic Dystrophy Pipeline Report
- DelveInsight’s Myotonic Dystrophy Pipeline analysis depicts a robust space with 22+ active players working to develop 20+ pipeline treatment therapies.
- The leading Myotonic Dystrophy Companies are working in the market include AMO Pharma, Harmony Biosciences, Dyne Therapeutics, Avidity Biosciences, Enzerna, Juvena Therapeutics, NeuBase Therapeutics, AMO Pharma Limited, Ionis Pharmaceuticals Inc, Expansion Therapeutics Inc, Myogem Health Company SL, Insmed Incorporated, Harmony Biosciences LLC, and others
- Promising Myotonic Dystrophy Pipeline Therapies in the various stages of development include Mexiletine, Tideglusib, rhIGF-I/rhIGFBP-3, DYNE-101, Supplement: MYODM, ERX-963, IONIS-DMPKRx, and others
- On February 2023, AMO Pharma Limited has announced drug named Tideglusib in the market by the phase 2 & 3. 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).
- On March 2023, Harmony Biosciences LLC has announced drug named Pitolisant Oral Tablet in the market by the phase 2. The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
- On March 2023, Lupin Ltd has announced drug named Mexiletine in the market by the phase 3. This is an open-label, multi-centre, single arm, interventional study to describe the steady-state PK, safety, and efficacy of mexiletine in paediatric patients (6 to
- On May 2023, AMO Pharma Limited has announced drug named Tideglusib in the market by the phase 1 2 & 3. This is an open-label phase 2/3 study for children and adolescents with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or children and adolescents with either Congenital or Childhood Onset DM1 who are treatment naïve.
- On June 2023, Dyne Therapeutics has announced drug named DYNE-101 in the market by the phase 1 & 2. 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).
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.
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Myotonic Dystrophy Emerging Drugs Profile
- Tideglusib: AMO Pharma
- Pitolisant: Harmony Biosciences
- DYNE-101: Dyne Therapeutics
Myotonic Dystrophy Pipeline Therapeutics Assessment
There are approx. 20+ Myotonic Dystrophy companies which are developing the therapies for Myotonic Dystrophy. The Myotonic Dystrophy companies which have their Myotonic Dystrophy drug candidates in the most advanced stage, i.e. phase II/III include, AMO Pharma.
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Myotonic Dystrophy Drugs and Companies
- Tideglusib: AMO Pharma
- Pitolisant: Harmony Biosciences
- AOC 1001: Avidity Biosciences
- DYNE-101: Dyne Therapeutics
- JUV 161: Juvena Therapeutics
- ENZ-001: Enzerna
Myotonic Dystrophy Therapeutics Assessment
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
Myotonic Dystrophy Market Drivers
- Technological advancements in Myotonic Dystrophy genomic research
- Management of multisystem disorder associated with Myotonic Dystrophy
Myotonic Dystrophy Market Barriers
- Lack of Approved therapies
- Lack of understanding of pathophysiology and mechanisms of DNA instability
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Scope of the Myotonic Dystrophy Pipeline Report
- Coverage- Global
- Myotonic Dystrophy Companies- AMO Pharma, Harmony Biosciences, Dyne Therapeutics, Avidity Biosciences, Enzerna, Juvena Therapeutics, NeuBase Therapeutics, AMO Pharma Limited, Ionis Pharmaceuticals Inc, Expansion Therapeutics Inc, Myogem Health Company SL, Insmed Incorporated, Harmony Biosciences LLC, and others
- Myotonic Dystrophy Pipeline Therapies- Mexiletine, Tideglusib, rhIGF-I/rhIGFBP-3, DYNE-101, Supplement: MYODM, ERX-963, IONIS-DMPKRx, and others.
- Myotonic Dystrophy Segmentation: Product Type, Molecule Type, Route of Administration
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Table of Content
- Introduction
- Executive Summary
- Myotonic Dystrophy: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Myotonic Dystrophy– DelveInsight’s Analytical Perspective
- Mid 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/II)
- DYNE-101: Dyne Therapeutics
- 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
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