Quest Magazine

SMN Gene Transfer Benefits Mice

A research group from the University of Sheffield in the United Kingdom has found that mice with a disease mimicking human spinal muscular atrophy (SMA) benefited significantly from intravenous transfer of the gene for the SMN (survival of motor neurons) protein. The mice lived significantly longer than untreated mice of the same type.

More Good News about Exon Skipping

AVI4658, an experimental treatment for patients with Duchenne muscular dystrophy (DMD) caused by certain mutations in the gene for the muscle protein dystrophin, has shown promising results when delivered intravenously to 19 trial participants.

Narrowing Down CMT Subtypes

 

Editor's note:This story was updated Feb. 4, 2011, to reflect the availability of a paper and editorial on this subject in Annals of Neurology.

Laid-Off Newsman with FSHD Starts Second Career

Peter Callas Jr. remembers as if it were yesterday the day his father gave him “the F.D.R. talk.”

It was 1973, and Peter Jr., then 13 years old, had just been diagnosed with facioscapulohumeral muscular dystrophy (FSHD).

Gentamicin Shows Mixed Results in DMD

Results of the MDA-supported trial of gentamicin in Duchenne muscular dystrophy (DMD) were presented Wednesday, April 14, at the annual meeting of the American Academy of Neurology (AAN), held in Toronto.

Levels of the needed dystrophin protein increased in six out of 12 participants who received the drug for six months. No functional improvements were seen.

Progress in Exon Skipping for DMD

The multinational pharmaceutical company GlaxoSmithKline (GSK) and the Dutch biotechnology company Prosensa  announced "intriguing results" following a 12-person trial of an experimental "exon skipping" therapeutic for Duchenne muscular dystrophy (DMD).

ALS Research: Zenvia Improves Emotional Symptoms in ALS

The experimental drug Zenvia, developed by Avanir Pharmaceuticals as a treatment for unwanted laughing/crying spells in ALS, has shown continued promise in lessening the frequency of such episodes in people with amyotrophic lateral sclerosis (ALS).

ALS Research: Zenvia Improves Emotional Symptoms in ALS

The experimental drug Zenvia, developed by Avanir Pharmaceuticals as a treatment for unwanted laughing/crying spells in ALS, has shown continued promise in lessening the frequency of such episodes in people with amyotrophic lateral sclerosis (ALS).

MDA Study Shows Lithium Not Helpful in ALS

An MDA-supported, 10-center trial of more than 100 people with amyotrophic lateral sclerosis (ALS) who took the oral medication lithium carbonate — some in conjunction with the ALS drug riluzole — has shown the drug does not provide benefit for disease progression or survival.

The study also showed that lithium can have concerning side effects, and when taken alone (without riluzole), may actually cause harm.

MDA Study Shows Lithium Not Helpful in ALS

An MDA-supported, 10-center trial of more than 100 people with amyotrophic lateral sclerosis (ALS) who took the oral medication lithium carbonate — some in conjunction with the ALS drug riluzole — has shown the drug does not provide benefit for disease progression or survival.

The study also showed that lithium can have concerning side effects, and when taken alone (without riluzole), may actually cause harm.

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