Inclusion-Body Myositis (IBM)

ALS, IBM — Benoit Coulombe, Ph.D.

Benoit Coulombe, director of the Proteomics and Gene Transcription Laboratory at the University of Montréal in Quebec, Canada, was awarded an MDA research grant totaling $377,067 over a period of three years to study the regulation of a protein whose gene, when mutated, can cause amyotrophic lateral sclerosis (ALS) and inclusion-body myositis (IBM).

Survey Open for People with IBM

A survey of people with inclusion-body myositis (IBM) is being conducted by A. David Paltiel, a professor of public health (health policy) and management at Yale University, with colleagues there and at the Myositis Association.

MD Briefs: Gene Therapy, Exon Skipping, Stem Cells

Update (Jan. 23, 2013):The "Building better utrophin" section was updated to reflect the availability of a Jan. 22, 2013, press release from the University of Missouri.

Below is a wrap-up of recent research news about the development of therapies for Duchenne, Becker and limb-girdle muscular dystrophies.

IBM — Virginia Kimonis

Virginia Kimonis, chief of the division of genetics and metabolism at the University of California, Irvine, received an MDA grant totaling $372,000 to conduct experiments designed to uncover the underlying molecular cause of inclusion-body myopathy associated with Paget's disease of Bone and/or frontotemporal dementia (IBMPFD).

IBM — Steven Greenberg, M.D.

MDA has awarded a grant totaling $444,314 over a period of three years to Steven Greenberg at Brigham and Women's Hospital and Harvard Medical School in Boston. The funds will help support Greenberg's study of the role of a protein called TDP43 in sporadic and hereditary inclusion-body myositis (IBM).

AAN Releases Guidelines for IVIG in Some Neuromuscular Diseases

The American Academy of Neurology (AAN) has released new guidelines on the use of a treatment called intravenous immunoglobulins (IVIG) in various neuromuscular disorders.

MDA 2012 Conference Report: Genetics and Immunology Update

More than 500 physicians, allied health care professionals and MDA staff attended the MDA's 2012 Clinical Conference in Las Vegas, March 4-7.

The program emphasized:

MDA Commits $12 Million to Neuromuscular Disease Research

The Muscular Dystrophy Association has awarded 38 new grants totaling more than $12 million to fund research projects focused on its continuing mission to uncover the causes of, and develop therapies for, the more than 40 neuromuscular diseases in its program.

MDA's Board of Directors reviewed and approved the new grants based on recommendations from the Association's Scientific and Medical Advisory Committees, and the grants took effect Feb. 1.

IBM - Sanford Bernstein, Ph.D.

Sanford Bernstein, a professor of biology at San Diego State University in California, was awarded an MDA research grant totaling $370,311 over a period of three years. The funds will help support Bernstein's research into the underlying molecular causes of, and potential treatments for, inclusion-body myopathy type 3 (IBM-3).

IBM-3 is caused by a mutation in a protein called myosin, the "molecular motor" that drives muscle contraction.

Seeking Participants for hIBM/GNE Clinical Trial

Update (Feb. 11, 2013): The phase 2 trial is no longer recruiting participants.