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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.

  • 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
  • BMS-986089 - Bristol-Myers Squibb's Candidate for Myostatin Inhibition
  • CARMESEAL-MD - Poloxamer 188 NF
  • CAT-1004 - Catabasis' MoveDMD Trial
  • CATENA®/RAXONE® - Phase 3 Study of Idebenone in Duchenne (DELOS)
  • 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 - Exon skipping Using Phosphorodiamidate Morpholino Oligomers (PMO) to produce functional dystrophin protein
  • EXON 2 SKIPPING - To induce Internal Ribosomal Entry Site (IRES) activation in Duchenne patients with Exon 2 Duplications
  • 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
  • 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-111Laminin-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
  • MYOBLAST TRANSPLANTATION - Myoblast Transplantation in Duchenne Patients
  • NBD Peptide - Using NF-ĸB blockers to Decrease Inflammation and Improve Muscle Function in Duchenne
  • PF-06252616 - Pfizer's Myostatin Inhibitor
  • RTC13 Read-Through Compound - Development of a drug that corrects nonsense mutations in patients with Duchenne
  • SMT C1100 - A Small Molecule Utrophin Modulator for Duchenne
  • SPIRONOLACTONE and EPLERENONE - Therapeutic Potential for Aldosterone Inhibition in 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 - Phase 3 Trial of Tadalafil for Duchenne
  • TAMOXIFEN - Using tamoxifen to improve muscle strength in Duchenne and Becker
  • TRANSLARNA (ataluren) - A New Drug for Nonsense Mutations by PTC Therapeutics
  • VBP15 - A Novel Steroid Customized for Duchenne

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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