Queen Square Centre for Neuromuscular Diseases


Hereditary Inclusion Body Myopathy-Patient Monitoring Program (HIBM-PMP): A Registry and Prospective Natural History Study to Assess HIBM Disease

Sponsor / Funder Ultragenyx
CI Professor Hanns Lochmüller
Sites Newcastle
Contact details Oksana.Pogoryelova@newcastle.ac.uk
More information


HIBM is a severe progressive myopathy. It typically starts in early adulthood as muscle weakness in the legs and progresses, ultimately leading to a wheelchair-bound state. The rate of progression is gradual and variable over the course of 10-20 years or longer.

There is a need to:

  • better understand the specific features of HIBM to improve awareness
  • help early diagnosis
  • identify patients
  • expand knowledge of the clinical presentation, progression and variation of the disease
  • identify and validate biomarkers and other outcome measures
  • inform on the design and interpretation of clinical trials of new drugs
  • and eventually to optimize patient management


Up to 10 centres in:

  • North America
  • European Union
  • and Middle East

Study information

HIBM Disease Registry is a database where patients can self-report their condition. Registry patients will not necessarily be associated with a clinical site.

Those registry patients who are in the same country as a study site may choose to have their data confirmed by one of those centres or opt-in to the HIBM Natural History Study.

Primary objective

To better understand HIBM.

HIBM Disease Registry's specific objectives:

  • Identify HIBM patients worldwide
  • Promote awareness and help diagnosis of HIBM disease in the neuromuscular field
  • Obtain an assessment of the medical history, clinical presentation and progression of disease in HIBM patients
  • Provide a connection for patients to the broader HIBM community and associated programs
  • Provide customized information to patients and their physicians that desire information on their disease status and progression.

HIBM Natural History Study's specific objectives:

  • Characterize HIBM disease presentation and progression over time using relevant clinical assessments
  • Obtain information using patient-reported outcomes (quality of life and timing of life changing events)