This year, I have been awed by the strength of our community. I think 2014 will be known as the year we effected change at every turn in clinical trials, on Capitol Hill, at the FDA, in doctors’ offices. And we did it as a community. Now it’s time to tackle the next challenge.

In 2015, PPMD will invest $600,000 to further the advancement of combination therapies in Duchenne. To start, we need to raise $400,000 this holiday season. That sounds big, I know, but thanks to our partners, it’s within reach—every gift today goes twice as far thanks to two Duchenne foundations that, like us, believe in the importance of combined therapies to treat this horrible disease.

Right now, we have multiple drugs that address different aspects of Duchenne advancing in clinical trials. We recognize that the best results will likely be achieved by using these drugs in combination. Combination therapeutics, after all, have been the most effective approach for diseases such as AIDS and cancer. In addition, combination therapies have the potential to benefit every person with Duchenne, regardless of mutation.

So we need to prepare now for the next stage of Duchenne research by systematically trying drugs together, first in animal studies and then in human trials. We also need to understand how regulators will view combination therapies and lay the groundwork now for rapid testing and approval of combinations.

Combination therapeutics are a crucial part of the fight to end Duchenne, and your gift will help us move this critical research forward. If we can raise $200,000 this month, we’ll reach the $400,000 we need thanks to generous matches from Rally for Ryan and Cure Dale’s Duchenne. We’re thrilled that these amazing foundations understand how collaborative effort will push forward our combination therapies.

We see strength when we combine forces voices, actions, dollars and work together. Your gift makes us stronger.

Please give right now, and have your donation doubled!

Warm regards,

Pat's Signature


P.S. Your online donation will be doubled thanks to the generous support of foundations that, like PPMD, believe in the idea of combination therapeutics to end Duchenne. Double your gift now!

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Comment by Ron Hill on December 4, 2014 at 5:01pm

NHS England does not currently routinely fund the use of Atularen (Translarna) for patients with Duchenne Muscular Dystrophy. However a new draft clinical commissioning policy covering its potential use has been developed in collaboration with a range of stakeholders, including patient and carer representatives, and taking into account the currently available evidence on clinical effectiveness. This draft policy continues to be one of a number of new draft policies being considered as part of NHS England's funding prioritisation process for 2015/16."

– NHS England
UNFORTUNATLY if you live in England,United Kingdom ... There is No possability getting any kind of Medication needed for DMD as the National Health Service is finding it hard to pay for these drugs BUT what is never considered is that it will cost more to pay for the longterm medical needs ... WE ARE FUCKED! 
Even in a medical emergency we can NOT use a Local Accident Emergency unit, because these units are staffed by jounior doctors that have No experience of the condition (DMD) and promtly take away Drugs like Bisoprolol (beta-blockers) because believe that the heart is beating too slow 70 bpm this is killing boy with DMD and they blame this in turn to DMD?
So for Safety We as Parents have to do all the thing that you would expect in A&E

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