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Myotonic Dystrophy Drug Pipeline Research Report 2023: Comprehensive Insights About 20+ Companies and 22+ Pipeline Drugs -

The "Myotonic Dystrophy - Pipeline Insight, 2023" drug pipelines has been added to's offering.

The "Myotonic Dystrophy - Pipeline Insight, 2023" report offers an extensive overview of the Myotonic Dystrophy pipeline landscape, featuring insights on over 20 companies and 22 pipeline drugs. It provides comprehensive profiles of these pipeline drugs, spanning both clinical and nonclinical stages, along with a thorough assessment based on product type, stage, route of administration, and molecule type.

Additionally, the report sheds light on inactive pipeline products in this domain. For a holistic understanding, it includes a detailed disease overview and Myotonic Dystrophy treatment guidelines. Furthermore, the report delves into a deep analysis of Myotonic Dystrophy from both commercial and clinical perspectives, providing valuable insights into drug mechanisms, clinical studies, NDA approvals, and product development activities, including collaborations, mergers, acquisitions, funding, and designations.

With a focus on novel approaches to Myotonic Dystrophy treatment, this report serves as a vital resource for industry players and researchers alike, presenting a comprehensive analysis of drugs in various clinical development stages, including phase II, I, preclinical, and Discovery, along with clinical trial details, pharmacological actions, collaborations, and the latest news and press releases in the field.

Myotonic Dystrophy Emerging Drugs

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, the activity of glycogen synthase kinase 3 beta (GSK3) has been shown to increase. AMO-02 is an inhibitor that has been shown to normalize levels of GSK3? in transgenic models and in ex vivo tissue samples in patients with DM1 and to reduce levels of the mRNA that is pathogenic for DM1. Currently, the drug is being investigated in the Phase II/III stage of clinical trial evaluation for the treatment of Congenital Myotonic Dystrophy.

Pitolisant: Harmony Biosciences

Pitolisant, which is being developed by Harmony Biosciences is being evaluated in the Phase II stage of its development for the treatment of myotonic dystrophy. 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.

DYNE-101: Dyne Therapeutics

DYNE-101 consists of an antigen-binding fragment antibody (Fab) conjugated to an antisense oligonucleotide (ASO) and is designed to enable targeted muscle tissue delivery with the goal of reducing toxic DMPK RNA in the nucleus, releasing splicing proteins, allowing normal mRNA processing and translation of normal proteins, and potentially stopping or reversing the disease.

Dyne has generated comprehensive preclinical data supporting its DM1 program, including reduction of nuclear foci and correction of splicing in patient cells, robust knockdown of toxic human nuclear DMPK RNA and correction of splicing in a novel in vivo model developed by Dyne, and reversal of myotonia in a disease model. In non-human primates, DYNE-101 demonstrated a favorable safety profile and achieved enhanced muscle distribution as evidenced by significant reduction in wild-type DMPK RNA.

In May 2023, Dyne Therapeutics announced that the European Medicines Agency (EMA) had granted orphan drug designation for DYNE-101.DYNE-101 is currently being evaluated in the Phase I/II global ACHIEVE trial, for people living with myotonic dystrophy type 1 (DM1).

Myotonic Dystrophy: Therapeutic Assessment

This segment of the report provides insights about the different Myotonic Dystrophy drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Myotonic Dystrophy

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

Key Questions Answered

  • How many companies are developing Myotonic Dystrophy drugs?
  • How many Myotonic Dystrophy drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Myotonic Dystrophy?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Myotonic Dystrophy therapeutics?
  • What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Myotonic Dystrophy and their status?
  • What are the key designations that have been granted to the emerging drugs?

Key Players

  • AMO Pharma
  • Harmony Biosciences
  • Dyne Therapeutics
  • Avidity Biosciences
  • Enzerna
  • Juvena Therapeutics
  • NeuBase Therapeutics

Key Products

  • Tideglusib
  • Pitolisant
  • DYNE-101
  • AOC 1001
  • ENZ-001
  • JUV 161
  • NT-0200


  • Late stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage product (Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration

Products have been categorized under various ROAs such as

  • Oral
  • Intravenous
  • Subcutaneous
  • Parenteral
  • Topical
  • Molecule Type

Products have been categorized under various Molecule types such as

  • Recombinant fusion proteins
  • Small molecule
  • Monoclonal antibody
  • Peptide
  • Polymer
  • Gene therapy
  • Product Type

Myotonic Dystrophy Report Insights

  • Myotonic Dystrophy Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs

Myotonic Dystrophy Report Assessment

  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs

For more information about this drug pipelines report visit

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