United States Myotonic Dystrophy Treatment Market 2031 | Growth Drivers, Key Players & Investment Opportunities

Market Size and Growth

Myotonic Dystrophy Treatment Market is growing with a High CAGR during the forecast period 2024-2031.

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Key Development:

Myotonic Dystrophy Treatment: Recent Developments

✅ In December 2025, Arrakis Therapeutics presented new preclinical in vivo data showing its orally available RNA‐targeted small molecule candidates for DM1 correct disease‐causing splicing defects and reverse myotonia in models, with plans to file an IND with the U.S. FDA in 2026.

✅ In November 2025, Sarepta Therapeutics provided a progress update on its investigational siRNA program SRP‐1003 for Myotonic Dystrophy Type 1 (DM1), reporting that its Phase 1/2 clinical trial continues to advance and enrollment at higher doses is ongoing with promising early indicators.

✅ In December 2025, the Muscular Dystrophy Association (MDA) announced more than $2.7 million in new research grants to accelerate breakthroughs across neuromuscular diseases, including Myotonic Dystrophy (DM), supporting discovery and early‐stage therapeutic research efforts.

✅ In November 2025, PepGen Inc. received a U.S. patent for its novel Myotonic Dystrophy Type 1 drug candidate PGN‐EDODM1, securing composition‐of‐matter protection that strengthens its development exclusivity and intellectual property position.

United States: Recent Myotonic Dystrophy Treatment Developments

✅ In November 2025, Avidity Biosciences reported that it has completed enrollment in its global Phase 3 HARBORTM trial of delpacibart etedesiran (del‐desiran) for DM1 and anticipates topline data in the second quarter of 2026, moving closer to potential regulatory filings.

✅ In October 2025, Avidity Biosciences announced that it completed a positive pre‐BLA meeting with the U.S. FDA regarding its upcoming biologics license application submission for delpacibart zotadirsen (del‐zota), setting the stage for further regulatory progress in 2026.

✅ In November 2025, Avidity Biosciences disclosed in its third quarter results that Novartis agreed to acquire the company for approximately $12 billion, unlocking strategic capital that may accelerate development of DM1 and other rare neuromuscular therapies.

✅ In September 2025, the ongoing global HARBOR Phase 3 trial of del‐desiran continued to enroll participants across multiple regions, reinforcing progress toward the first potential approved therapy for DM1 in the U.S. and globally.

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Japan: Recent Myotonic Dystrophy Treatment Developments

✅ In November 2025, patient advocacy groups in Japan highlighted that international collaborative clinical trials for DM1 (including the HARBOR study) are actively involving Japanese sites, expanding domestic access to cutting‐edge investigational treatments.

✅ In October 2025, the Remudy patient registry updated activities and hosted educational webinars on myotonic dystrophy, reflecting growing research engagement and community involvement in Japan.

✅ In August 2025, discussions at a Japanese neuromuscular event emphasized the importance of advancing global Phase 3 DM1 trial participation and local research support structures, strengthening Japan's role in treatment development networks.

✅ In July 2025, the national focus on rare disease research included reporting on clinical and research forums about myotonic dystrophy, underlining Japan's ongoing commitment to supporting treatment development and patient engagement.

Key Players:

=> Dyne Therapeutics, Teva Pharmaceuticals, Ani Pharmaceuticals, AA Pharma, Alkem Laboratories Ltd among others.

Emerging Players
=> Vertex Pharmaceuticals, Avidity Biosciences, PepGen, and among others.

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Key Segments:

By Disease Type

DM1 (Type 1 Diabetes Mellitus) - ~35% share

Predominantly affecting pediatric and young adult populations; market driven by pain management related to diabetic neuropathy.

DM2 (Type 2 Diabetes Mellitus) - ~65% share

Largest segment due to high prevalence in adults; increasing demand for oral and injectable therapies to manage neuropathic pain.

By Drug Class

Sodium Channel Blockers - ~20% share

Used to control nerve hyperexcitability; common for diabetic neuropathy and chronic pain management.

Tricyclic Antidepressants (TCAs) - ~15% share

Widely prescribed for neuropathic pain; adoption influenced by cost-effectiveness and efficacy in DM1 and DM2 patients.

Benzodiazepines - ~10% share

Used primarily for anxiety and sleep disorders associated with diabetes; moderate market presence.

Calcium-antagonists - ~10% share

Adopted for pain modulation and cardiovascular comorbidities; growth in DM2 patients with neuropathy.

Analgesics - ~30% share

Largest segment; includes NSAIDs and other pain-relief medications widely used in DM1 and DM2 populations.

Others - ~15% share

Includes novel therapies under clinical trials and combination therapies targeting diabetic neuropathy.

By Route of Administration

Oral - ~55% share

Most preferred route due to convenience and patient compliance; dominant in DM2 patients.

Injectable - ~30% share

Includes subcutaneous or intravenous formulations; widely used in DM1 and advanced DM2 cases.

Topical - ~15% share

Creams, patches, and gels for localized neuropathic pain management; growing adoption for mild to moderate cases.

By Distribution Channel

Hospital Pharmacies - ~40% share

Primary channel for injectable and high-cost therapies; significant in DM1 patient care.

Retail Pharmacies - ~45% share

Dominant channel for oral therapies and analgesics; convenient access for chronic DM2 patients.

Online Pharmacies - ~15% share

Increasing adoption due to telemedicine and e-pharmacy growth; higher convenience for urban and remote populations.

By Region

North America - ~35% share

High prevalence of DM1 and DM2; strong R&D, healthcare infrastructure, and patient access to advanced therapies.

Europe - ~25% share

Moderate growth; driven by aging population, rising DM2 prevalence, and established healthcare systems.

Asia-Pacific - ~30% share

Fastest-growing market; increasing DM2 prevalence, growing healthcare access, and rising adoption of oral therapies.

South America - ~5% share

Emerging market; demand primarily from DM2 patients with rising urbanization and lifestyle changes.

Middle East & Africa - ~5% share

Nascent market; gradual growth from DM2 patient population and increasing healthcare awareness.

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Phone: +1 877 441 4866
Website: https://www.datamintelligence.com

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