What Research?
Research
Problem
protein Isolated in Muscular Dystrophy
Scientists
may have just taken an important step in the fight against Muscular Dystrophy
.According to the Journal of Clinical Investigation, researchers at
Gene Therapy 2006
As an 8-year-old boy
becomes the first
Possible stem Cell
treatment 2008
News article on the
potential treatment of Duchenne Muscular Dystrophy with Mesoangioblasts, a type
of stem cell, that hopefully would be harvested from the patient.
Preliminary results from Exon skipping trial announced
This refers to Duchene rather than Becker but is an important trial as you may read below
The exon skipping technique tries
to change a Duchenne mutation into a Becker mutation. If a variation
disturbs the reading frame and thus causes Duchenne dystrophy, the reading
frame can be restored by artificially removing one or more exons directly in
front or after the deletion, the duplication, or the exon which contains a
point mutation. The result would be to change the severe Duchenne symptoms into
the much milder symptoms of Becker muscular dystrophy.
Prof. Dominic Wells, Imperial College London, member of the MDEX consortium said:
“The results of the first MDEX clinical trial shows that exon-skipping works without significant side effects in patients treated locally (injected into a muscle in the foot) with the molecular patch produced in collaboration with AVI Biopharma. We look forward to starting the intravenous trial in the first quarter of 2009 which will show if body wide treatment is possible and safe.”
On February 3 2009, PTC Therapeutics announced that they have completed the patient enrollment for participation in phase IIb of their clinical trial of PTC124™, two months ahead of schedule.
See the full item on this subject on the Muscular Dystrophy’s site:
Click here to go to the MDC’S Research page
See also the Research page on the MDA’s site
Click here to goto the MDA’s page
