The myotonic dystrophy market is expected to grow significantly due to the advancements in treatment options, greater healthcare access, and a rising prevalence of the condition, which together foster higher demand for innovative and effective therapies such as Tideglusib (AMO-02; AMO Pharma), Delpacibart etedesiran (formerly AOC-1001; Novartis/Avidity Biosciences), NaMuscla (mexiletine; Lupin), and others.

LAS VEGAS, Sept. 23, 2026 /PRNewswire/ -- Recently published Myotonic Dystrophy Market Insights report includes a comprehensive understanding of current treatment practices, myotonic dystrophy emerging drugs, market share of individual therapies, and current and forecasted market size from 2022 to 2036, segmented into leading markets [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].

Myotonic Dystrophy Market Summary

- The market size for myotonic dystrophy was found to be USD 236 million in the 7MM in 2025.

- The United States accounted for the largest myotonic dystrophy treatment market size in the 7MM in 2025, compared to other major markets, including the EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan.

- In 2025, the aggregate number of individuals diagnosed with myotonic dystrophy across the 7MM was approximately ~110,000.

- Leading myotonic dystrophy companies, such as AMO Pharma, Novartis, Avidity Biosciences, Lupin, Dyne Therapeutics, PepGen, Vertex Pharmaceuticals, Sarepta Therapeutics, ARTHEx Biotech, Juvena Therapeutics, Modalis Therapeutics, and others, are developing new myotonic dystrophy treatment drugs that can be available in the myotonic dystrophy market in the coming years.

- The promising myotonic dystrophy therapies in clinical trials include Tideglusib (AMO-02), Delpacibart etedesiran (formerly AOC-1001), NaMuscla (mexiletine), Zeleciment basivarsen (z-basivarsen), PGN-EDODM1, VX-670, SRP-1003, ATX-01, JUV-161, MDL-202, and others.

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Key Factors Driving the Growth of the Myotonic Dystrophy Market

- Rising Disease Awareness and Diagnosis: Growing awareness among patients and healthcare professionals is improving recognition of myotonic dystrophy. Increased access to genetic testing and specialized neuromuscular clinics is also supporting earlier and more accurate diagnosis.

- Increasing Adoption of Genetic Testing: Advances in molecular diagnostics are making genetic testing more accessible and reliable for confirming myotonic dystrophy. Greater use of genetic counseling and family screening is expanding the diagnosed patient population.

- Advancements in Disease-Modifying Therapies: The emergence of antisense oligonucleotides, RNA-targeted therapies, and other precision approaches is creating opportunities beyond supportive care. These innovations are expected to expand the treatment landscape and market potential.

- Increasing Research and Clinical Trial Activity: Greater understanding of the molecular mechanisms underlying myotonic dystrophy is accelerating drug discovery and clinical development. Growing numbers of clinical trials of therapies such as Tideglusib (AMO-02; AMO Pharma), Delpacibart etedesiran (formerly AOC-1001; Novartis/Avidity Biosciences), NaMuscla (mexiletine; Lupin), Zeleciment basivarsen (z-basivarsen; Dyne Therapeutics), and others are strengthening the pipeline.

Aparna Thakur, Project Manager of Forecasting & Analytics at DelveInsight, said that if upcoming candidates demonstrate durable functional benefits, treatment adoption, diagnosis rates, and payer willingness to reimburse premium therapies are likely to increase, supporting long-term market growth.

Myotonic Dystrophy Market Analysis

- Currently, there are no approved therapies capable of curing myotonic dystrophy (DM) or slowing its underlying disease progression.

- Consequently, treatment primarily focuses on symptomatic management to alleviate disease burden and improve patients' quality of life.

- Among the various manifestations of DM, myotonia is one of the most commonly treated symptoms.

- Antimyotonic agents, including mexiletine, lamotrigine, carbamazepine, oxcarbazepine, flecainide, propafenone, phenytoin, and ranolazine, may be used for symptom management, although these therapies are prescribed off-label.

- The pharmacological management of chronic muscle pain generally follows a stepwise approach based on the WHO analgesic ladder, adapted for chronic non-cancer pain.

- Treatment commonly begins with nonsteroidal anti-inflammatory drugs (NSAIDs), with adjunctive therapies introduced as required. These may include anticonvulsants, antidepressants, muscle relaxants, and topical treatments such as lidocaine or capsaicin patches.

- The myotonic dystrophy pipeline includes emerging therapies such as AMO-02 from AMO Pharma, mexiletine from Lupin, and delpacibart etedesiran (del-desiran) from Novartis and Avidity Biosciences.

- In addition, preclinical development includes RNA-targeted small-molecule (rSM) candidates, such as the program being developed by Arrakis Therapeutics.

Myotonic Dystrophy Competitive Landscape

Some of the myotonic dystrophy drugs under development include Tideglusib (AMO-02; AMO Pharma), Delpacibart etedesiran (formerly AOC-1001; Novartis/Avidity Biosciences), NaMuscla (mexiletine; Lupin), Zeleciment basivarsen (z-basivarsen; Dyne Therapeutics), PGN-EDODM1 (PepGen), VX-670 (Vertex Pharmaceuticals), SRP-1003 (Sarepta Therapeutics), ATX-01 (ARTHEx Biotech), JUV-161 (Juvena Therapeutics), MDL-202 (Modalis Therapeutics), and others.

AMO Pharma's AMO-02 is an orally administered small-molecule inhibitor of glycogen synthase kinase 3β (GSK3β) being developed for the treatment of congenital myotonic dystrophy. Preclinical and clinical studies have indicated its potential to restore GSK3β levels and reduce pathogenic mRNA associated with myotonic dystrophy type 1 (DM1). The therapy has received several regulatory designations from the US FDA, including Orphan Drug Designation (ODD), Fast Track Designation (FTD), and Rare Pediatric Disease (RPD) designation.

Del-desiran, Novartis/Avidity's lead Antibody Oligonucleotide Conjugate (AOC), is designed to address the underlying molecular cause of myotonic dystrophy type 1 by reducing disease-associated DMPK mRNA. The therapy consists of a TfR1-targeting monoclonal antibody conjugated to a small interfering RNA (siRNA). It has received Breakthrough Therapy Designation (BTD), Fast Track Designation (FTD), and Orphan Drug Designation (ODD) from the US FDA, as well as orphan designation in the European Union, underscoring its potential as a treatment for DM1.

The anticipated launch of these emerging therapies are poised to transform the myotonic dystrophy market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the myotonic dystrophy market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.

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Recent Developments in the Myotonic Dystrophy Market

- In September 2026, Dyne Therapeutics, Inc. announced that it will present additional one-year clinical data from the Phase 1/2 ACHIEVE trial evaluating zeleciment basivarsen (z-basivarsen, also known as DYNE-101) in patients with myotonic dystrophy type 1 (DM1). The data will be presented at the 31st Annual International Congress of the World Muscle Society (WMS), taking place virtually and in Hiroshima, Japan, from September 29 through October 3, 2026, and at the 2026 Annual Meeting of the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM), scheduled to be held virtually and in Orlando, Florida, from September 29 through October 2, 2026.

- In September 2026, Novartis announced that its investigational treatment, delpacibart etedesiran, failed to achieve the primary endpoint in a Phase III clinical trial for myotonic dystrophy type 1.

- In July 2026, AMO Pharma Limited announced that it has reached an agreement with regulatory authorities in the U.S., U.K., and Canada regarding the design of a registrational clinical study to evaluate the safety and efficacy of its investigational therapy, AMO-02 (oral tideglusib), for the treatment of congenital myotonic dystrophy type 1 (cDM1).

- In May 2026, PepGen Inc. announced that data from its PGN-EDODM1 program were presented in a poster and late-breaking oral presentation at the 15th International Myotonic Dystrophy Consortium (IDMC-15), held May 26–30, 2026, in Saguenay, Quebec, Canada.

- In March 2026, ARTHEx Biotech announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to ATX-01 for the treatment of myotonic dystrophy type 1 (DM1).

Myotonic Dystrophy Epidemiology Segmentation

The myotonic dystrophy epidemiology section provides insights into the historical and current myotonic dystrophy patient pool and forecasted trends for the leading markets. Based on DelveInsight's estimates, in 2025, the diagnosed prevalent population of myotonic dystrophy in the United States was estimated to be approximately 57,000.