Clinical Research

MD Center Clinical Research Projects 2022

This is a brief overview of active and upcoming studies at the University of Minnesota. A full list and description of all clinical trials is available on, as required by U.S. Law.

**Feel free to contact our Clinical Research Coordinators for more information on these research studies**

Research for Any Individual:

  • Dr. James Ervasti: Outcome Measures for Neuromuscular Disease

    • Evaluate muscle loss overtime in individuals with neuromuscular conditions through analysis of metabolites found in urine.
  • U of MN Muscular Dystrophy Registry
    • The purpose of this research study is to collect existing and future information onyou and/or your family member with the goal of determining natural history and clinical effectiveness of healthcare services in patients with neuromuscular disease.
  • MDA Registry
    • The goals of the MOVR Data Hub are:
      • Improve Clinical Care
      • Accelerate Therapy Development
      • Enhance Understanding of Disease

Research for DMD:

  • Astellas: A Randomized, Double-Blind, Placebo-Controlled Phase 1b Study with Open-Label Extension to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Patients with Duchenne Muscular Dystrophy (DMD)
    • To evaluate the safety and tolerability of ASP0367 as well as its efficacy on muscle function. 
    • Ages 8-16 year old males with genetic confirmation of DMD
  • Sarepta Therapeutics Registry: A Long-term Observational Study Evaluating Sarepta Therapeutics, Inc.’s Exon-Skipping Therapies in Patients with Duchenne Muscular Dystrophy under Conditions of Routine Clinical Practice 
    • To describe the usage, and potential benefits and risks of long-term use of eteplirsen or golodirsen in patients with Duchenne muscular dystrophy in routine clinical practice
    • No age restriction. Must be receiving eteplirsen or golodirsen medication or plan to start the medication within 6 months of enrollment.
  • Dr. Peter Karachunski: Imaging in DMD
    • To investigate dystrophic changes of muscle involvement in patients with DMD using imaging techniques such as MRI and ultrasound
    • To validate use of ultrasound for screening and monitoring purposes in patients with DMD during clinical studies and trials
    • Ages 10-30 year old males with genetic confirmation of DMD

Research for SMA:

  • AveXis registry: A Prospective, Long-Term Registry of Patients with a Diagnosis of Spinal Muscular Atrophy (SMA) – (RESTORE).
    • To assess the long-term outcomes of patients with a diagnosis of SMA and also characterize and assess long-term safety and effectiveness of AVXS-101.
    • To assess overall survival of all patients with SMA.
    • No age restriction. Must have genetic confirmation of SMA on or after 24 May 2018.

Research for FSHD:

  • Dr. Michael Kyba: Tissue biopsies for FSHD study
    • To determine whether a molecular or cellular defect can be attributed to cells of FSHD muscle by comparing to those of unaffected individuals
    • Genetic confirmation of FSHD
    • Ages 4+ (muscle biopsy for >18 years of age ONLY)
    • FSHD + Control to participate

Research for DM1:

  • Harmony: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients with Myotonic Dystrophy Type 1, Followed by an Open-label Extension.
    • To evaluate the safety and efficacy of pitolisant compared with placebo in treating EDS in patients with DM1
    • Ages 18-65 with genetic confirmation of myotonic dystrophy type 1
  • Dr. Georgios Manousakis: Serum uric acid as biomarker of type I myotonic dystrophy
    • This is a pilot study aimed to determine whether there is any potential role of uric acid (UA) in the pathophysiology of myotonic dystrophy type 1 (DM1)
    • Ages 18-85 with genetic confirmation of myotonic dystrophy type 1

The list of Clinical Trials available is ever growing. For the most up-to-date list of Muscular Dystrophy Clinical Trials and resources, please go to to see all trials hosted by the U of MN MD Center.