Quest Magazine

IBM Research: Doubts about Tau

Abnormal accumulation of a protein called "tau" has been considered by many to contribute to muscle degeneration in inclusion-body myositis (IBM). But recently, MDA grantee Steven Greenberg and colleagues at Brigham and Women's Hospital and Harvard Medical School in Boston have cast doubt on this purported disease mechanism and say it's too early to develop drugs for IBM based on it.

Advocacy: Catching Legislators at Home

Thanks to a new communications effort by MDA’s Advocacy Department – the Fly Out -- members of Congress are getting to hear the concerns of the MDA community first-hand and on their home turf.

The MDA Fly Out, which has been taking place around the country throughout August, capitalizes on Congress’ annual recess when most legislators head to their home districts and meet with constituents, either one-on-one or at public meetings.

Abnormal Activation

An MDA-supported team of scientists in the United States and the Netherlands has uncovered new leads about the origins of facioscapulohumeral muscular dystrophy (FSHD), a disease whose biochemical underpinnings have proved elusive to scientists despite years of investigation.

Lack of understanding of the mechanisms involved in FSHD has impeded treatment development, a phase of research that generally moves forward after disease mechanisms have been described.

Doubts about Tau

Abnormal accumulation of a protein called "tau" has been considered by many to contribute to muscle degeneration in inclusion-body myositis (IBM). But recently, MDA grantee Steven Greenberg and colleagues at Brigham and Women's Hospital and Harvard Medical School in Boston have cast doubt on this purported disease mechanism and say it's too early to develop drugs for IBM based on it.

Come Fly Out with Me

Thanks to a new communications effort by MDA’s Advocacy Department – the Fly Out – members of Congress are getting to hear the concerns of the MDA community first-hand and on their home turf.

The MDA Fly Out, which has been taking place around the country throughout August, capitalizes on Congress’ annual recess when most legislators head to their home districts and meet with constituents, either one-on-one or at public meetings.

MG: Out of Place

Stepped-up production of fragments of acetylcholine receptors, the microscopic "landing pads" on muscle fibers that normally help process signals from the nervous system, may provoke the immune system and worsen myasthenia gravis (MG), new research shows.

In mice with an MG-like disease, these receptor fragments (which are proteins) are abundant but nonfunctional, and they end up in the wrong place in muscle fibers.

Out of Place

Stepped-up production of fragments of acetylcholine receptors, the microscopic "landing pads" on muscle fibers that normally help process signals from the nervous system, may provoke the immune system and worsen myasthenia gravis (MG), new research shows.

In mice with an MG-like disease, these receptor fragments (which are proteins) are abundant but nonfunctional, and they end up in the wrong place in muscle fibers.

Meds Access: A Clearer Path

The U.S. Food and Drug Administration (FDA) announced Aug. 12, 2009, that it has launched a Web site to help doctors and patients apply to obtain medications that have not yet been approved by the FDA ("investigational" drugs).

Rescuing SBMA-Affected Muscles

A protein known as insulin-like growth factor 1 (IGF1) may provide a new lead in the treatment of spinal-bulbar muscular atrophy (SBMA), also known as Kennedy disease.

A multinational team coordinated by MDA research grantee Maria Pennuto at the Italian Institute of Technology in Genoa, has found that having extra IGF1 genes seems to improve muscle strength and function in mice bred to have an SBMA-like disease.

A Clearer Path

The U.S. Food and Drug Administration (FDA) announced Aug. 12, 2009, that it has launched a Web site to help doctors and patients apply to obtain medications that have not yet been approved by the FDA ("investigational" drugs).

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