Neuromuscular Research

Our research program includes cutting edge investigations into the pathogenetic mechanisms underlying various neuromuscular disorders, biomarkers to facilitate early diagnosis, and potential therapeutic target/s.

Basic Research at UChicago

  1. Drosophila models of axonal degeneration and ALS (Grace Zhai, PhD) (link to profile & publications)
  2. Pathogenetic mechanisms of SOD1 and C9ORF72-associated ALS (Raymond Roos, MD) (link to profile & publications)
  3. Pathogenetic mechanisms in inflammatory neuropathies and myasthenia (Betty Soliven, MD)(link to profile & publications)
  4. Congenital myasthenia and cerebellar ataxias (Christopher Gomez, MD PhD)(link to profile and publications. 

Of note, University of Chicago has established the Center for Motor Neuron Disease (co-Directors: Dr. Paschalis Kratsios and Dr. Rong Zhai) that includes faculty colleagues in our Neuroscience Institute who are investigating molecular mechanisms pertinent in motor neuron development and degeneration. https://neuroscience.uchicago.edu/center-motor-neuron-disease

Clinical Research & Trials 

Previous and ongoing PI-initiated studies:

  1. Intermuscular coherence as a biomarker for upper motor neuron involvement in ALS (co-PIs: Kourosh Rezania, MD, Naoum Issa, MD, PhD)
  2. Immunologic studies in myasthenia and autoimmune neuropathies (PI: Betty Soliven, MD)

Recently completed clinical trials sponsored by pharma: 

  1. A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients with Generalized Myasthenia Gravis. 

    Site PI: Betty Soliven, MD

  2. A Phase 3, Randomized, Double-blind, Placebo-controlled study evaluating efficacy and safety of rozanolixizumab in adult patients with generalized myasthenia gravis.

       Site PI: Kourosh Rezania, MD

  1. A Phase 3, randomized, Double-blind, Placebo-controlled multicenter study to evaluate efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab in patients with generalized myasthenia gravis.

    Site PI: Betty Soliven, MD

     Ongoing and upcoming clinical trials sponsored by pharma: 

  1. A randomized, double-blind, placebo-controlled phase III study to evaluate the efficacy, safety, and tolerability of iptacopan in patients with generalized Myasthenia Gravis (gMG), followed by an open-label extension phase.

    Site PI: Kourosh Rezania, MD; status- enrolling

  2. A randomized, double-blind, placebo-controlled phase III study to evaluate the efficacy, safety, and tolerability of remibrutinib in patients with generalized Myasthenia Gravis, followed by an open-label extension phase.

    Site PI: Kourosh Rezania, MD; status- pending

  3. A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T cells (CABA-201) in Participants with Generalized Myasthenia Gravis. 

    Site PI: Kourosh Rezania, MD; status-pending

  4. Healey Platform Trial for the Treatment of Amyotrophic Lateral Sclerosis (ALS): A perpetual multi-center, multiregimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS

    Site PI: Kourosh Rezania, MD; status- awaiting new regimen

  5. A Phase 2b, Multi-center, Randomized, Double-blind, Placebo controlled study of IMVT-1402 Treatment in Adult Participants with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

    Site PI: Betty Soliven, MD; status- pending