Sunday, November 25, 2012

New drug to be tested for use in the muscular dystrophies

thursday 22 November 2012
Muscular Dystrophy Campaign
Landon.



New drugs to be tested for use in muscular dystrophies

   Prof Dominic Wells, and Dr Susan Brown, at the Royal Veterinary College in London recently received a grant from the Medical Research Council (MRC) as part of an innovative scheme that saw the MRC partnering with pharmaceutical company AstraZeneca.   AstraZeneca made 22 of its chemical compounds available free-of-charge to scientists, who were encouraged to apply for MRC funding to use them in medical research with the ultimate aim of benefiting patients. AstraZeneca had conducted early trials of these compounds and validated their use for future research, but had put them on hold for further development. The aim of this partnership was to extend the possible application of these compounds for use in new areas.



       Prof Wells and Dr Brown will be testing a drug called AZD1236 for its ability to prevent scar tissue formation in muscular dystrophy.  AZD1236 was designed to block the activity of an enzyme called matrix metallopeptidase-9 (MMP-9) which is known to be involved in the formation of scar tissue. In the muscular dystrophies, there is a gradual breakdown of the muscle which leads to muscle weakness.  As the muscle breaks down it is replaced by fat and scar tissue further reducing the ability of the muscles to work properly. It is this process that the researchers hope to slow using AZD1236.

        The researchers will use the drug, originally developed by AstraZeneca to treat a type of lung disease known as chronic obstructive pulmonary disease, in two different mouse model of muscular dystrophy - Duchenne muscular dystrophy and limb girdle muscular dystrophy to see if they can slow progression of the disease. If successful, the study will provide the evidence they need to plan human trials of the drug to test whether it has a benefit for individuals with muscular dystrophy.

Wednesday, November 7, 2012

Research news (phase 1 exon skipping trial )




GlaxoSmithKline releases results of phase 1 exon skipping trial 

Tuesday 6 November 2012


The drug company GlaxoSmithKline has today announced results of a phase 1 clinical trial of a potential exon skipping drug -now called drisapersen - in boys with Duchenne muscular dystrophy who are unable to walk. This drug, a molecular patch for exon 51 of the dystrophin gene, has previously been tested in boys with Duchenne muscular dystrophy who are still able to walk and has generated encouraging results. The main aim of this trial was to make sure there are no additional safety issues for wheelchair users and to test whether different doses of the potential exon skipping drug were safe and well tolerated.

    The company recruited 20 boys who were given one of three doses of drisapersen or a placebo (an inactive substance). The participants received a single injection of the drug and were monitored for up to 6 months. All the boys who received the potential drug experienced some mild side effects such as redness or swelling of the injection site and all the boys given the highest dose of the potential drug experienced fever. However, the researchers say no serious side effects were observed.

Tuesday, November 6, 2012

hydrotherapy for muscular dystrophy (mayopathy)





aquatic therapy for DMD 
(MAYOPATHY-TAMILNADU-INDIA)


                            WATER SURVIVAL SESSION




                          POSTURE TRAINING SESSION




                                  DIVE START SESSION