Stand Up For Science

This is a report of the start of clinical trials of a new
drug for the treatment of Duchenne Muscular Dystrophy (DMD), a
progressive disease that causes wasting and atrophy of muscle tissue.

The novel drug uses an antisense oligonucleotide (AO) patch to mask the
faulty part of the Dystrophin gene that stops production of the
dystrophin protein. These "nonsense" mutations are responsible for about
15% of DMD cases, and it is hoped that masking the fault will prevent
the development of DMD in young patients who have not yet lost their
muscle function.

Animal research, particularly that using the the mdx mouse model of DMD,
made a vital contribution to the basic research that lead to this
treatment, and to its later evaluation prior to human trials. It is
hoped that this AO drug will be safe and effective in DMD patients,
though it will be several years before we know whether or not it
prevents the development of DMD.

http://news.bbc.co.uk/1/hi/health/7052453.stm
http://www.muscular-dystrophy.org/news/mdcled.html
http://www1.imperial.ac.uk/medicine/research/researchthemes/neuroscience/mol_mechanisms/cmntransgen/

Paul Browne