Pediatric Neuromuscular Research

Here at Lurie Children’s, our research team works tirelessly to bring our patients and families an abundance of clinical research opportunities, including cutting-edge gene therapy clinical trials. Our multidisciplinary team of clinicians and research staff conduct various research projects to help further the groundbreaking change to treatments for children with neuromuscular disorders. We participate in studies funded by the National Institute of Health (NIH), pharmaceutical companies and our own investigator-initiated studies. We hope our research efforts will improve the lives for children and families living with neuromuscular disorders.

Research Team

Current Neuromuscular Clinical Trials

Natural History of Clinical, Biochemical, Pathologic and Genetic Markers in Children with Neuromuscular Weakness

  • IRB # 2011-14701
  • Study Contact: Hannah Munson,
  • PI: Dr. Nancy Kuntz
  • Description: The purpose of this study is to collect natural history, biochemical, pathologic and genetic markers in individuals with neuromuscular weakness. This study offers the potential value for improving clinical care of patients and will aid in designing future clinical trials. 

Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Expanded Access Program (EAP) of Idebenone in Patients with Duchenne Muscular Dystrophy

Study of SRP-4045 and SRP-4053 in DMD Patients (ESSENCE)

An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (IGNITE DMD)

A Phase 3 Open-Label Study of Eculizumab in Pediatric Participants With Refractory Generalized Myasthenia Gravis (gMG)

INCEPTUS: A Prospective, Non-Interventional Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects Aged 3 Years and Younger

A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects (INCEPTUS)

Study of Intrathecal Administration of AVXS-101 for Spinal Muscular Atrophy (STRONG)

Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 (STR1VE)

A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies. (SHINE)

A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy (NURTURE)

A Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-onset Spinal Muscular Atrophy (TOPAZ)