MSU and Orphazyme: Working together for a treatment for inclusion body myositis

As part of our mission at Myositis Support and Understanding Association (MSU), we are constantly looking for ways to advocate for you, the Myositis Community, by engaging with research and pharmaceutical companies to better the lives of patients and caregivers. One of our goals is to provide you with access to research.

To this end, MSU is excited to announce a new relationship with Orphazyme, the Danish biopharmaceutical company sponsoring the Arimoclomol Phase II/III clinical trial for sporadic inclusion body myositis (sIBM).

In a series of conference calls with Regan Sherman, Associate Director of Patient Advocacy Relations, we discussed the various ways Orphazyme and MSU can work together to benefit those living with sporadic inclusion body myositis. These talks were very productive and we look forward to realizing many of the ideas and events discussed.

As a part of our relationship, we will bring information and updates directly to the sIBM patient and caregiver communities. We will also work together with Orphazyme to help guide education, awareness, outreach, and other efforts valuable to our membership.

“This is an exciting mutually beneficial relationship with a shared goal of a successful clinical trial and ultimately, a treatment for sIBM patients,” says Jerry Williams, Founder and President of Myositis Support and Understanding Association.

Many patients are interested in the arimoclomol trial and with good reason. Sporadic inclusion body myositis (sIBM) is a rare, disabling inflammatory and muscle-wasting disease, one of several in the group of muscle diseases called the Idiopathic Inflammatory Myopathies. There are currently no available treatment options for patients with sIBM. Rather, patients are usually directed to utilize exercise to help slow disease progression by maintaining muscle strength and to make life adaptations, or life hacks, to better live with this disabling disease.

There are currently six (6) clinical trial locations in the U.S. and one (1) in the UK, with more coming over the next few months. The newest location announced is the University of Rochester in NY.

See full Arimoclomol trial information

Further clinical trial summary information can be found on the Pipeline tab on the Orphazyme website.

As more locations are up and running, we will be sharing the information with you on our website, Facebook, Twitter, Instagram, and via email updates. If you have not yet, we invite you to register for your free membership with MSU and to follow us on our social networking sites.

Register for your free MSU membership

We thank Regan and the entire Orphazyme team for their willingness to work with and learn from myositis nonprofits and community organizations. We also thank the inclusion body myositis community for their strength, patience, and excitement as we continue to work for them.

As with any relationship MSU enter into with pharmaceutical companies, our policies will not be determined or guided by any contributions, financial or otherwise. Member privacy is always protected and members private information is never shared with any third parties unless expressly consented, including informed and signed consent.

About Arimoclomol

Arimoclomol is a new chemical entity with a very favorable safety and tolerability record in humans: Seven Phase I clinical studies have been conducted in healthy volunteers. Arimoclomol is administered orally, three times daily, and can be easily dissolved in liquids or food for best possible patient comfort and compliance. Orphazyme has obtained orphan drug designation for arimoclomol in NPC, ALS, and sIBM from both the FDA and EMA.

About Orphazyme A/S

Orphazyme is a Danish biotech company listed on Nasdaq Copenhagen with a late-stage drug pipeline, developing new treatment options for orphan protein-misfolding diseases. The company was founded in 2009 based on early scientific discovery in heat-shock proteins. The company is headquartered in Copenhagen and currently has 30 employees. The lead candidate arimoclomol is in development as a potential treatment for four orphan diseases; two neuromuscular diseases, sporadic Inclusion Body Myositis (“sIBM”) and Amyotrophic Lateral Sclerosis (“ALS”), and two lysosomal storage diseases, Niemann Pick Type C (“NPC”) and Gaucher disease. For more information, please visit



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.

View more information: Myositis Support


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