Registered charity
number 1146355

The Gift of Hope

 

Duchenne Now trustees recently presented a proposals to our members, Isofen. Duchenne Now members voted to financially support this trial that shows hope and promise as a treatment for this generation living with Duchenne.

 

All we want is a treatment for Duchenne... This could become an exciting reality...

 

Isofen is being funded by Duchenne Now and other International DMD foundations. This appeal is about taking the funding to completion and making 2014 the the year of action not words.

Every penny donated to the "Gift of Hope" campaign will go directly to funding Isofen.

 

www.justgiving.com/giftofhope

Give the gift of hope and make a donation today to make hope an exciting reality for this generation living wih Duchenne. 

 

ISOFEN

ISOFEN is novel therapeutic approach for Duchenne Muscular dystrophy based on two drugs, ibuprofen, a non steroidal anti-inflammatory agent, and isosorbide dinitrate, a drug that releases nitric oxide.

ISOFEN has passed successfully all dafety tests also in humans and will be now investigated for efficacy in humans, In particular it will be investigated whether ISOFEN slows the progression of Duchenne Muscular dystrophy in non ambulant patients. The study will have a total duration of 24 months of which 6 for the enrolment of patients and 18 for the actual trial, in which the primary outcome measure of efficacy will be the amelioration of the Motor Function Measure scale.

The mechanisms by which ISOFEN operates has been established in animal models of dystrophy; essentially ISOFEN enhances muscle repair and myogenesis, reduces inflammation and adipose tissue deposition and enhances muscle function. It also prevents the exhaustion of the myogenic stem cell pool. this results in a significant amelioration of the disease in mouse models.

These actions do not address the genetic cause of the disease, as instead occurs in the case of exon skipping, but act downstream on key common pathogenic mechanisms. Because of this, if proven successful in non-ambulant patients, ISOFEN can be easily transferred to ambulant patients.

If ISOFEN is proven successful it will be authorized for use in patients in a few months after completion of the trial, since this trial is agreed with regulatory Agencies and all preclinical and phase I studies (healthy volunteers) have been done. In essence we expect that the drug will become available by February 2016.

The total cost for the study is approximately 1485000 US $ of which 500000 have already been raised

To discuss these projects in further detail please contact the Duchenne Now office on 01706 693399.

Support & Advice

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Supplements and medications

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Adaptations

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Educational needs and benefits

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Wheelchairs and equipment

Fundraising

Every single penny raised for Duchenne Now will go to fund Duchenne research. How will you help?  Click here for more info

Membership

Donating regularly by standing order is one of the best ways in which you can become a part of and support the work of Duchenne Now.  Click here for more info

Events

We hold plenty of events to raise money to fund research. Why not join us at the next one to show your support?  View our calendar