Quest Magazine

Caution: Immune Response Seen in DMD Gene Therapy

Unwanted responses by the immune system to dystrophin have been seen in a small, MDA-supported clinical trial of gene therapy for Duchenne muscular dystrophy (DMD)— an unexpected finding, investigators say.

Rather than a setback, the finding is “the beginning of a new way of thinking” about gene therapy, said Jerry Mendell, director of the Center for Gene Therapy at Nationwide Children's and a long-time MDA research grantee and MDA-associated clinician. Mendell was the neurologist on this trial.

DMD/BMD: Taking a Closer Look at Ataluren

PTC Therapeutics, a South Plainfield, N.J., biopharmaceutical company, has announced findings that reflect the company's closer look at a large-scale trial of its experimental drug ataluren.

The additional results, presented April 16 at the American Academy of Neurology  meeting in Toronto, show that trial participants who took the lower dose of ataluren did better on a six-minute walking test than did participants who took the placebo or higher dose.

Luring Away Myostatin Can Boost Muscle Size

ACE031, a laboratory-modified protein developed by Acceleron Pharma of Cambridge, Mass., has shown promise as a therapy to increase muscle mass, based on results of a trial in healthy volunteers. The company will now test it in Duchenne muscular dystrophy (DMD).

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).

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