Duchenne Muscular Dystrophy Early access to medicines and treatments

Those are the words of Daniel Baker, 38, from the Reddings, who suffers from Duchenne Muscular Dystrophy (DMD), a disease which causes muscle degeneration. If complications occur with the respiratory system, it can sometimes prove fatal.

But Daniel is hoping a cure can be found in this lifetime, even if there might only be “a small chance”.

The Government agreed earlier this year that new medicines and treatments which could treat serious illnesses will be fast-tracked to patients, after years of campaigning by a Gloucestershire man, Les Halpin.

Les, from Tetbury, was a motor neurone disease sufferer who has fought for years for early access. He died last September at the age of 56.

Early access to medicines and treatments could help Cheltenham man with Duchenne Muscular Dystrophy.

Early access to medicines and treatments could help Cheltenham man with Duchenne Muscular Dystrophy.

The new scheme will allow those with seriously debilitating conditions the lifeline of trying new medicines years before they go through the normal licensing process.

One of those companies who will be looking to apply is Summit, a pharmaceutical company developing treatments for DMD.

The company’s lead drug has finished phase one studies in healthy volunteers and it is now being tested on DMD boys in four hospitals in UK. The next study will start later this year, something Daniel said he would volunteer for.

He said: “I hope this is the start of something new for medicine, not just DMD, but any life-threatening illness. I understand the need for trials, but really when you’re facing something life limiting, it’s worth the risks, as long as the patient agrees and is made fully aware that something could go drastically wrong.

“I would volunteer, I understand the risks involved, but sometimes the end result is worth taking that risk. I’m doubtful anything would cure me but just stopping the progression is enough.

“I have heard of the next wonder drug since I was a teen. There have been many drugs that have failed, but everyone has gone through long periods of testing, so maybe early access can speed up the science by seeing what works in humans and what does not.”

Diagnosed when he was just three, he started using a wheelchair when he was 11, before his lungs starting failing after completing university.

Since then he has had to wear a ventilator everyday.

“I hope for a cure, but don’t hold out any false hope. There’s only a small chance I will see one in my lifetime, and I have no illusions that I’ll be able to walk again, my body has come too far for that,” Daniel said.

“At most, I’ll get some strength back and not need my ventilator, possibly even be able to chew and swallow solid foods, again. It would be amazing just to do that.

“If there isn’t a cure, I’ll just fight on as I always have. Any life is better than no life.”

He hopes a cure will be found soon, so children with DMD can live a better life.

“There have been many recent breakthroughs and encouraging test results.

“Being optimistic, I think we will see treatments in the next decade or so, if not cures.

“I would hope the researches could learn from me and I am happy to help in any way.

“I intend to have a muscle biopsy soon, I doubt anything shown will help me, but maybe a scientist will pick it up and find something useful.

“Some limits should be placed on experiments but we live in a culture so scared of being sued we are going backwards.

“People are becoming scared of science as we only hear the bad stories. We need to get the excitement of science back.

“The moon landings, for example, would never have happened in this century. There were huge risks, and people have died, but the choice was theirs, and they became heroes and encouraged a generation of people to achieve so much.”

Tagged on: ,

Leave a Reply

Your email address will not be published. Required fields are marked *

You may use these HTML tags and attributes: <a href="" title=""> <abbr title=""> <acronym title=""> <b> <blockquote cite=""> <cite> <code> <del datetime=""> <em> <i> <q cite=""> <strike> <strong>