Quest Magazine

FA Research: Idebenone Not Effective

On May 19, 2009, Santhera Pharmaceuticals reported that a phase 3 trial of its idebenone compound Catena showed the drug was not associated with a statistically significant benefit in 70 children between 8 and 17 years old with Friedreich's ataxia who took it for six months. (See the company's press release, Santhera's US Phase III IONIA Trial in Friedreich's Ataxia Misses Primary Endpoint.)

ALS Research: Survival Gene

A variant version of the gene for a protein known as KIFAP3 has been found to increase survival time in people with sporadic (nonfamilial) ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease) by an average of 14 months.

ALS Research: Survival Gene

A variant version of the gene for a protein known as KIFAP3 has been found to increase survival time in people with sporadic (nonfamilial) ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease) by an average of 14 months.

The findings of John Landers at the University of Massachusetts Medical School in Worcester, with colleagues from institutions around the world, was published online May 18, 2009, in Proceedings of the National Academy of Sciences. MDA supported Orla Hardiman and Simon Cronin at the Royal College of Surgeons in Dublin, Ireland, for this work.

DMD, MG Research: Spotlight on Prednisone

Some interesting findings about prednisone’s effect on behavior in DMD, and on drugs that may alter its usage in myasthenia gravis, were part of the 61st annual meeting of the American Academy of Neurology (AAN), held recently in Seattle.

DMD STUDY

Daily prednisone led to better behavior than weekly, high-dose prednisone

CMT Research: Gene Benefits Mice

Zarife Sahenk at Nationwide Children's Hospital and Ohio State University in Columbus, and colleagues, found mice with a disease resembling type 1A Charcot-Marie-Tooth disease (CMT) benefited from a transfer of genes for the neurotrophin 3 protein. CMT1A is caused by a duplication of the PMP22 gene.

Jerry Mendell, who has received many MDA research grants and co-directs the MDA clinic at Nationwide Children's, was part of the study team, as was Brian Kaspar, who has MDA support at Nationwide.

Searching for Signs

The 61st annual meeting of the American Academy of Neurology (AAN), held in Seattle April 25-May 2, included several ALS-related presentations.

Searching for Signs

The 61st annual meeting of the American Academy of Neurology (AAN), held in Seattle April 25-May 2, included several ALS-related presentations.

Stem Cells to Nerve Cells

Researchers at the Burnham Institute for Medical Research in La Jolla, Calif., and the University of California at Los Angeles, say they've developed immature nerve cells that are flexible enough to become multiple nervous-system cell types but committed enough not to become other types of cells or form tumors.

ALS: A Vicious Cycle

A vicious cycle in which damage to nerve cells (neurons) in the spinal cord results in the loss of an important mechanism to protect neurons, causing more neuron loss, has been identified as a possible contributor to ALS (amyotrophic lateral sclerosis). Identification of this pathway opens the door to targeting it with therapeutic agents.

ALS: A Vicious Cycle

A vicious cycle in which damage to nerve cells (neurons) in the spinal cord results in the loss of an important mechanism to protect neurons, causing more neuron loss, has been identified as a possible contributor to ALS (amyotrophic lateral sclerosis). Identification of this pathway opens the door to targeting it with therapeutic agents.

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