Clinical Trials
Advancing Care, Shaping Tomorrow

Below is a list of key contacts for Duchenne Muscular Dystrophy (DMD) clinics and clinical trials, along with steps to sign up. I’ve organized this into clinics, trials, and advocacy groups to simplify your search:

Finding Duchenne Muscular Dystrophy Clinics

1. DMD Specialized Clinics (Contact Directly)
These clinics offer multidisciplinary care (neurology, cardiology, physical therapy, etc.) for DMD patients.

United States

  • MDA Care Centers
    • Website: MDA Care Center Locator
    • Phone: 1-833-ASK-MDA1 (1-833-275-6321)
    • How to sign up: Use the locator tool, then contact the clinic directly for appointments.
  • Nationwide Children’s Hospital (Columbus, OH)
  • Kennedy Krieger Institute (Baltimore, MD)
    • Neuromuscular Clinic: Website
    • Phone: (443) 923-9400
  • Boston Children’s Hospital
    • Neuromuscular Center: Website
    • Phone: (617) 355-2756

International

  • Great Ormond Street Hospital (London, UK)
    • Neuromuscular Service: Website
    • Phone: +44 20 7405 9200
  • Sydney Children’s Hospital (Australia)
    • Neuromuscular Clinic: Website
    • Phone: +61 2 9382 1111

2. Clinical Trials (How to Join)

To sign up for DMD trials, follow these steps:

Step 1: Search for Trials

  • ClinicalTrials.gov
    • Search: DMD Trials (use keywords: Duchenne muscular dystrophy)
    • Filter by location, recruitment status (e.g., “Recruiting”), and age group.
  • TREAT-NMD Global Registry
    • Website: TREAT-NMD → Links to country-specific trial registries.
  • CureDuchenne

Step 2: Check Eligibility

  • Each trial lists inclusion/exclusion criteria (e.g., age, mutation type, ambulatory status).
  • Work with your neurologist or clinic to confirm eligibility.

Step 3: Contact the Trial Coordinator

  • Use the contact information listed on the trial’s page (often an email or phone number).
  • Example: A trial at Nationwide Children’s Hospital may list:

Step 4: Submit Pre-Screening Forms

  • Trials often require medical records, genetic reports, and a referral from your doctor.

3. Advocacy Groups (Personalized Help)

These organizations help connect families to clinics/trials:

  • Parent Project Muscular Dystrophy (PPMD)
    • Website: ParentProjectMD.org
    • Phone: 1-800-714-5437
    • Services: Trial matching, clinic referrals, and family support.
  • Muscular Dystrophy Association (MDA)
    • Website: MDA.org
    • Phone: 1-833-ASK-MDA1
    • Services: Care center referrals and trial updates.
  • World Duchenne Organization

Key Notes

  • Eligibility: Trials often target specific mutations (e.g., exon-skipping therapies).
  • Costs: Some trials cover travel expenses; ask the coordinator.
  • Timing: Trials may take months to start—sign up for newsletters from advocacy groups for updates.

For immediate assistance, call PPMD or MDA—they’ll guide you through the process!

To find clinics or events related to Duchenne Muscular Dystrophy (DMD), here are actionable steps and resources:

1. Treatment Clinics (Medical Care):

  • MDA Care Centers:
    • The Muscular Dystrophy Association (MDA) has a network of specialized clinics. Use their Care Center Locator to find facilities offering multidisciplinary DMD care.
  • TREAT-NMD Global Registry:
    • For international options, visit TREAT-NMD to access neuromuscular care directories.

2. Research Clinics/Trials:

  • ClinicalTrials.gov:
    • Search for ongoing or upcoming DMD trials using keywords like “Duchenne” here. Filter by location or status (recruiting, active).

3. Educational Events & Workshops:

  • Parent Project Muscular Dystrophy (PPMD):
    • Check their events calendar for webinars, conferences (e.g., Annual Connect Conference), and family workshops.
  • World Duchenne Organization:
    • Explore global awareness events and educational resources here.

4. Local Institutions:

  • Contact renowned neuromuscular centers like:
    • Boston Children’s Hospital (US)
    • Johns Hopkins Hospital (US)
    • Nationwide Children’s Hospital (US)
    • Great Ormond Street Hospital (UK)
  • Universities or hospitals near you may host DMD clinics—check their websites or call directly.

5. Online Communities:

  • Join forums like Duchenne Connect or social media groups (Facebook, Reddit) for real-time updates from families and advocates.

Notes:

  • Real-Time Data: This information is based on pre-2023 knowledge. Verify details through the provided links, as schedules and availability change.
  • Personalized Assistance: Reach out to organizations like MDA (1-833-ASK-MDA1) or PPMD for personalized help locating clinics/events.

By leveraging these resources, you can identify both medical care clinics and educational events tailored to DMD. Always confirm event dates/trial statuses directly with the providers.

Duchenne Muscular Dystrophy (DMD) is a severe genetic disorder characterized by progressive muscle degeneration. Clinical trials are essential for developing new treatments and improving patient outcomes. Below is an overview of notable clinical trials and therapeutic developments in DMD:​

1. Vamorolone (Agamree): Vamorolone is a dissociative steroid that selectively binds to the glucocorticoid receptor, exerting anti-inflammatory and immunosuppressive effects. In a 48-week randomized controlled trial, vamorolone at a dose of 6 mg/kg/day demonstrated maintenance of improvement in motor outcomes and significant improvement in linear growth compared to prednisone. The U.S. Food and Drug Administration (FDA) approved vamorolone for treating DMD in October 2023. â€‹en.wikipedia.org

2. Givinostat (Duvyzat): Givinostat is a histone deacetylase inhibitor with potential anti-inflammatory and muscle-preserving properties. An 18-month phase III study showed that participants treated with givinostat experienced a statistically significant slower decline in the time to climb four stairs compared to placebo. The FDA approved givinostat for medical use in March 2024, making it the first nonsteroidal medication approved to treat all genetic variants of DMD. â€‹en.wikipedia.org+2en.wikipedia.org+2en.wikipedia.org+2

3. Eteplirsen (Exondys 51): Eteplirsen is an antisense oligonucleotide designed to induce exon 51 skipping during mRNA processing, leading to the production of a functional dystrophin protein. Clinical studies demonstrated increased dystrophin production in participants, and in September 2016, the FDA granted accelerated approval for eteplirsen in the United States. However, the European Medicines Agency denied its approval in Europe. â€‹de.wikipedia.org+1en.wikipedia.org+1

4. SGT-003: SGT-003 is an experimental gene therapy developed by Solid Biosciences for DMD. It is considered an advancement over the company’s earlier gene therapy, SGT-001. While specific clinical trial data for SGT-003 is limited, it represents ongoing efforts to improve gene therapy approaches for DMD. â€‹en.wikipedia.org

5. Ezutromid: Ezutromid is an orally administered small molecule aimed at modulating utrophin, a protein similar to dystrophin. Despite initial promise, a Phase 2 clinical trial was terminated in 2018 after ezutromid failed to show efficacy in slowing disease progression. â€‹en.wikipedia.org

6. Elevidys (SRP-9001): Elevidys is a gene therapy developed by Sarepta Therapeutics. In January 2025, late-stage trial data showed positive results after two years of treatment in male patients aged four or older with DMD. The therapy significantly reduced difficulties in standing, walking, and running, with improvements increasing between one and two years post-treatment. The safety profile remained consistent, with no new safety signals identified. â€‹investors.com+2Time+2wsj.com+2wsj.com+1Time+1

7. WVE-N531: Wave Life Sciences reported positive interim data from a mid-stage trial of WVE-N531, an investigational treatment for DMD. The study showed substantial dystrophin production in patients, with the treatment being safe and well-tolerated. The company is seeking accelerated approval and anticipates further data by Q1 2025. â€‹marketwatch.com+1investors.com+1

For comprehensive and up-to-date information on ongoing and upcoming clinical trials for DMD, consider the following resources:

  • Parent Project Muscular Dystrophy (PPMD): Provides a detailed overview of current clinical trials and research studies related to DMD. â€‹parentprojectmd.org
  • CureDuchenne: Offers a table of currently enrolling DMD clinical trials involving active drugs, updated as of January 2025. â€‹cureduchenne.org
  • DMD Hub’s Clinical Trial Finder: Aggregates trustworthy information on existing and upcoming DMD trials in the UK. â€‹DMD Hub

These resources can assist patients, families, and healthcare providers in identifying suitable clinical trials and understanding the evolving landscape of DMD research.

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