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Duchenne Therapeutic Pipeline

Therapeutic Pipeline

There are many different therapeutic strategies underway development to treat Duchenne—the “Therapeutic Pipeline. The following clinical trial or research FAQ sheets are family-friendly summaries of actively recruiting clinical trials and research studies. Many are for pre-clinical research that is soon to be in clinical trial.

These FAQ sheets are written for the program book of the PPMD Annual Connect Conference (held every June).  We house them on DuchenneConnect since they are a useful resource for families and professionals. Although we try to include the majority of studies in the United States, this is not intended to be a comprehensive list.

Click on the name of any therapeutic below to read more about it specifically, or review a summary of the major Research Strategies for Duchenne or an overview of the Drug Development Process. You can also view the latest Research News.

  • AAV Microdystrophin Gene Therapy - Gene Therapy AAV Delivery of Microdystrophin to Restore Muscle Protein Expression in Duchenne
  • ACCESS DMD™ - Deflazacort Early Access Program
  • ARM210 - Using ARM210 to Improve Muscle Strength & Function in Duchenne
  • AT-300 - Akashi’s Novel Modulator of Stretch-Activated Calcium Channels
  • BECKER MUSCULAR DYSTROPHY - A Natural History Study to Predict Efficacy of Exon Skipping
  • BIGLYCAN - A Unique Utrophin Upregulator
  • BMB-D001 - Mini-dystrophin Gene Therapy for Duchenne
  • BMS-986089 - Bristol-Myers Squibb's Investigational Candidate for Myostatin Inhibition
  • CARMESEAL-MD - Poloxamer 188 NF
  • CAT-1004 - Catabasis' MoveDMD Trial
  • COENZYME Q10 AND LISINOPRIL- Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
  • COUGH in DUCHENNE and BECKER - Peak Cough Flow and Cough Clearance in Duchenne and Becker Muscular Dystrophy
  • DEFLAZACORT - A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescents with Duchenne
  • DP ARF ULTRASOUND - Double Push Acoustic Radiation Force Ultrasound for Monitoring Degeneration
  • DT-200 - A Selective Androgen Receptor Modulator to Improve Muscle Strength & Function in Duchenne
  • DUCHENNE MUSCULAR DYSTROPHY - A Longitudinal Study of the Natural History of Duchenne
  • ETEPLIRSEN - Study 4658-203 for Early Stage Duchenne
  • ETEPLIRSEN - Study 4658-204 for Advanced Stage Duchenne
  • ETEPLIRSEN - Study 4658-301/PROMOVI
  • EXON 2 SKIPPING - To induce Internal Ribosomal Entry Site (IRES) activation in Duchenne patients with Exon 2 Duplications
  • FG-3019 - An Investigational Therapeutic Monoclonal Antibody to inhibit the activity of Connective Tissue Growth Factor (CTGF)
  • FOLLISTATIN GENE TRANSFER - Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne
  • FOLLISTATIN GENE TRANSFER - Follistatin Gene Transfer to Patients with Becker Muscular Dystrophy
  • FOR-DMD - Finding the Optimal steroid Regimen for Duchenne Muscular Dystrophy
  • GALGT2 Gene Therapy - Viral gene transfer for GALGT2 as a surrogate gene therapy for Duchenne
  • GENE TRANSFER OF MICRO-DYSTROPHIN - Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin
  • Genetic Modifiers - Genetic Modifiers of Duchenne and Becker Muscular Dystrophy
  • GIVINOSTAT (ITF2357) - A Histone Deacetylase (HDAC) Inhibitor for the treatment of Duchenne
  • HOPE - Halt cardiOmyopathy ProgrEssion in Duchenne
  • HT-100 - Akashi's Phase 1/2 Clinical Program in Duchenne
  • ImagingDMD - Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
  • iPS CELL THERAPY - Induced Pluripotent Stem Cells for Duchenne
  • ISOFEN 3 - A combination drug for the treatment of Duchenne
  • LAMININ-111 - Laminin-111, Integrin and Utrophin as a Potential Therapy for Duchenne
  • MICROSOFT BAND - A Potential Outcome Measure for Boys with Duchenne
  • MRI and CARDIOPULMONARY FUNCTION - Using MRI to Assess Cardiopulmonary Function in Duchenne
  • MTB-1 - MTB-1 mediated gene regulation
  • MYOBLAST TRANSPLANTATION - Myoblast Transplantation in Duchenne Patients
  • NBD Peptide - Using NF-ĸB blockers to Decrease Inflammation and Improve Muscle Function in Duchenne
  • NS-065/NCNP-01 for Exon 53 Skipping - Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne
  • PF-06252616 - Pfizer's Myostatin Inhibitor
  • PhaseOut DMD - A 48 week Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with Ezutromid (Formerly Known as SMT C1100) in Ambulatory Boys with Duchenne
  • RAXONE® - Phase 3 Study Assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid steroids (SIDEROS)
  • RAXONE® - Phase 3 Study of Idebenone in Duchenne Muscular Dystrophy (DELOS)
  • RTC13 Read-Through Compound - Development of a drug that corrects nonsense mutations in patients with Duchenne
  • SPIRONOLACTONE and EPLERENONE - Therapeutic Potential for Aldosterone Inhibition in Duchenne
  • SRP-4045/SRP-4053 (ESSENCE/4045-301) - A 96 -Week, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients with Duchenne
  • STEROIDS IN YOUNG BOYS - Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne
  • STRENGTH TRAINING - Development of a Strength Training Protocol in Duchenne
  • TADALAFIL - A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy
  • TAMOXIFEN - Using tamoxifen to improve muscle strength in Duchenne and Becker
  • TRANSLARNA (ataluren) - A New Drug for Duchenne Muscular Dystrophy Caused by a Nonsense Mutation
  • VAMOROLONE - A Potential Steroid Alternative Customized for Duchenne
  • Wave Life Sciences – Exon Skipping Medications
  • WELLSTONE MRI/MRS BIOMARKERS - Failed Regeneration in the Muscular Dystrophies

For a look at some of the therapeutics Parent Project Muscular Dystrophy are funding, visit our Funding Portfolio.

If you have any questions about the FAQ sheets, please email

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