Update on the Clinical Studies Using Arimoclomol

presented by Dr. Mazen Dimachkie, MD, FAAN, FANA

The ongoing arimoclomol clinical trial for sporadic inclusion body myositis (sIBM) continues to generate a lot of questions from our community. As part of our commitment to providing the latest information on sIBM research and clinical trials, we asked Dr. Mazen Dimachkie, Principal Investigator for the Phase II/III Arimoclomol Studies and Professor of Neurology and Director of the Neuromuscular Division at University of Kansas Medical Center, if he would provide us with an update.

Dr. Dimachkie was gracious enough to take the time to talk with Jerry Williams and Lynn Wilson. The presentation and Q&A were based on the sIBM community feedback and questions about the trial.

Watch the video below

Download the slides

Dr. Dimachki has provided us with a copy of the slides used during his presentation. Download for future reference.

View and Download the slides

What is IBM?

Learn more about inclusion body myositis (IBM) and find resources and support for IBM patients and caregivers.

What is IBM?

Who is Orphazyme A/S?

Read more about Orphazyme, a 2019 MSU sponsor, and our collaboration to help find a treatment for IBM.

Orphazyme and MSU

About Mazen M. Dimachkie, MD, FAAN, FANA

Professor of Neurology & Director of Neuromuscular Division
Executive Vice Chairman & Vice Chairman for Research Program, Department of Neurology
University of Kansas Medical Center, Kansas City, Kansas, USA

Mazen DimachkieMazen M. Dimachkie, MD, FAAN, FANA, received his undergraduate and medical degrees from the American University of Beirut in Lebanon before completing 2 years of Internal Medicine training at Saint Agnes Hospital in Baltimore, Maryland. Dr. Dimachkie then completed a residency in Neurology at UT-Houston from July 1, 1990 to June 30, 1993 and fellowship in electromyography & Neuromuscular Disease from July 1, 1993 to June 30, 1994, both at The University of Texas Health Science Center at Houston (UT-Houston). He is ABPN board-certified in Neurology, Clinical Neurophysiology, and Neuromuscular Medicine and holds a UCNS certificate in Clinical Neuromuscular Pathology. He was elected as fellow member of the American Academy of Neurology and of the American Neurological Association. After being on faculty at the UT-Houston Neurology Department 1995-2007, he was recruited to lead the prolific neuromuscular Section and now Division at the University of Kansas Medical Center in Kansas City (KUMC). He is now Professor of Neurology, director of the Neuromuscular Division at the KUMC, director of the Neuromuscular Medicine Fellowship and Executive Vice Chairman & Vice Chairman for Research at the Neurology Department. As the director of KUMC Neuromuscular Research, he manages one of the busiest neuromuscular clinical trials units in North America. He leads and participates in a wide variety of federally-funded neuromuscular research studies and industry-sponsored studies, nationally and internationally. He has authored or co-authored more than 400 abstracts, articles, and book chapters. He is a frequently invited lecturer in the fields of myositis, myasthenia gravis, ALS, neuropathies and other neuromuscular topics. Dr. Dimachkie serves on the executive committee of the Muscle Study Group, and serves on the MSABs of the Myasthenia Gravis Foundation of America and The Myositis Association.

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Myositis Support and Understanding Association (MSU) is a patient-centered, all-volunteer 501(c)(3) nonprofit organization Empowering the Myositis Community. Founded by Myositis patients, for Myositis patients, MSU provides education, support, advocacy, access to research and clinical trial matching, and need-based financial assistance.

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  1. Louise Samantha Jane Green 3 years ago

    The video was very good and hopeful. Yet it was indicated by Jerry Williams two months ago as having been a negative study. Does this mean arimoclomol has been ruled out as a treatment. Is rapamycin being considered anywhere for the treatment of IBM?

  2. Jerry Williams 3 years ago

    Unfortunately, it was a negative study and has been ended. We are working hard to move forward to the next opportunities to help find a treatment for IBM.

  3. Louise Samantha Jane Green 3 years ago

    When will arimoclomol be available as a treatment for IBM in the UK? My father has suffered from it for 10 years now, getting progressively worse and is now unable to walk without a frame and can only get up from a raised chair. His consultant had heard of the drug but did not realise it could be a treatment for IBM.

    Thank you.

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