Five Strategies for Treating Neuromuscular Disease

Antisense oligonucleotides block flawed genetic instructions

Antisense oligonucleotides — also called antisense, oligos, or simply AONs— are pieces of genetic code that keep other genetic code from being processed. Designed to pair up with a particular sequence of DNA or RNA, AONs can change, block or destroy targeted genetic instructions in a variety of ways.

Researchers Exploring Disability Perceptions

Researchers at the Psychology of Disability Lab at the University of Michigan in Ann Arbor are exploring the social identity of people with disabilities through a short, anonymous, Web-based questionnaire.

The lab's Disability Identity Project is being headed by principal investigator Adena Rottenstein, a doctoral candidate in psychology.

The study closes the week of Aug. 22, 2011.

Love Letters and Preimplantation Genetic Diagnosis

It was the worst Monday morning of our lives — and Mondays are universally bad.

My wife, Monique, and I woke up to a snow lockdown in London. The snow had started gently enough in the evening, but now on this January morning it was a white strait jacket. We could hardly move, but we desperately needed to get to Nottingham at all costs. Our potential future children — two fertilized and rapidly growing embryos — lay in wait for us in a Nottingham fertility clinic almost three hours away by train.

MDA Conference Brings Together Researchers, Clinicians, Industry

Moving therapeutic strategies from the laboratory to clinical trials and ultimately to the market as treatments was the theme of the MDA National Scientific Conference held March 13-16, 2011, in Las Vegas.

Some 300 people attended the conference, the first in a planned series of such MDA-sponsored meetings that will emphasize new research and current medical care. The majority of presenters and many of the audience members were current or former MDA research grantees or physicians at MDA-supported clinics.

Study Seeks People With Uncertain MD Diagnoses

Not One But Two DNA Changes Are Needed to Cause FSHD

Facioscapulohumeral muscular dystrophy (FSHD) requires the presence of not one but two genetic changes, both on chromosome 4, before it causes its characteristic symptoms — weakness starting in the muscles of the face, shoulder blade area and upper arms, with possible progression to other parts of the body.

The new findings, announced online Aug. 19, 2010, in the journal Science, have immediate implications for diagnosis and prediction of FSHD, and possible long-term implications for its treatment.

MDA Awards Two New FSHD Grants

MDA has awarded two grants for research aimed at determining the precise molecular causes of facioscapulohumeral dystrophy (FSHD), and developing therapies for the disease.

MDA and Friends of FSH Research (FFSHR) based in Kirkland, Wash., will jointly fund a two-year, $200,000 grant to Joel Chamberlain, an assistant professor of medical genetics at the University of Washington in Seattle.

Am I Disabled or Aren't I Disabled?

While I lay on a plinth, a Swiss doctor gestured that I should lift up my left leg. He bear-hugged my leg and resisted its elevation with his full body weight. He grunted as he fought my movement.

He wasn’t the only person prodding and inspecting me. A team of doctors, biokineticists and physiotherapists surrounded me. It was like the United Nations of medics. Besides the Swiss doctor, there was a lady from Israel, a young athletic Austrian and two Americans.

Research Administration Winter 2009

Updated MD-CARE Act is now law

The Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education (MD-CARE) Amendments of 2008 received final Congressional approval on Sept. 27 and were signed into law by President Bush Oct. 8.

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