Quest Magazine

Drug Used to Treat Sepsis Shows Promise in Mice with ALS

A compound known as "activated protein C" (APC) that is already in use to treat severe bloodstream infections also may have benefit in ALS, according to a recently published report.

DMD Research: 'Remarkable' Results

Mice with a severe disease closely resembling human Duchenne muscular dystrophy (DMD) have responded extremely well to a new "exon skipping" compound that targets the specific dystrophin gene error these mice have.

Researchers noted "a remarkable prevention of the dystrophic pathology and improvement of the muscle function" in these severely affected mice, which lack both the dystrophin and utrophin muscle proteins. (Mice in previous exon skipping trials lacked only dystrophin, the protein missing in DMD.)

A Promising New Development for Treatment of Duchenne Muscular Dystrophy

A technique called exon skipping shows great potential to increase muscle strength and prolong life in people with a severe form of Duchenne muscular dystrophy (DMD). According to a study that included MDA-supported Stephen Wilton at the University of Western Australia, exon skipping improves production of a crucial muscle protein that’s missing in people with DMD. For the first time, these results were observed in mice with an especially severe form of muscular dystrophy.

Exon Skipping Proves Effective in Mice with a Severe DMD-Like Disease




Stephen Wilton

ALS Research: Blocking SOD1

A phase 1 clinical trial of the experimental drug ISIS-SOD1-Rx in patients with the SOD1-related type of familial (inherited) amyotrophic lateral sclerosis (ALS) is expected to begin before the end of 2009 at Washington University in St. Louis, Massachusetts General Hospital in Boston and four additional U.S. sites.

DMD Research: Drug Company On Board

On Oct. 13, 2009, the multinational pharmaceutical company GlaxoSmithKline announced it will develop and commercialize the experimental “exon skipping” compound PRO051 for Duchenne muscular dystrophy (DMD).

The commitment of a major pharmaceutical company to development of a drug for DMD is very good news for families with this disease.

ALS Research: Blocking SOD1

A phase 1 clinical trial of the experimental drug ISIS-SOD1-Rx in patients with the SOD1-related type of familial (inherited) amyotrophic lateral sclerosis (ALS) is expected to begin before the end of 2009 at Washington University in St. Louis, Massachusetts General Hospital in Boston and four additional U.S. sites.

ALS TDI Webcast

A Webcast is now available of the fourth annual research symposium held as part of the ALS TDI Leadership Summit 2009 on Oct. 5. The symposium was conducted at the MDA-supported ALS Therapy Development Institute in Cambridge, Mass.

ALS TDI Webcast

A Webcast is now available of the fourth annual research symposium held as part of the ALS TDI Leadership Summit 2009 on Oct. 5. The symposium was conducted at the MDA-supported ALS Therapy Development Institute in Cambridge, Mass.

Trial of SOD1 Blocker in Familial ALS to Open Soon

A phase 1 clinical trial of the experimental drug ISIS-SOD1-Rx in patients with the SOD1-related type of familial (inherited) amyotrophic lateral sclerosis (ALS) is expected to begin before the end of 2009 at Washington University in St. Louis, Massachusetts General Hospital in Boston and four additional U.S. sites.

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