At the start of Cystic Fibrosis Awareness Week, REBECCA TAYLOR meets a family affected by the disease and finds out about the prospects for sufferers.
AFTER years of research the UK's 7,500 Cystic Fibrosis (CF) sufferers could be helped by a scientific breakthrough that may stop the disease.
Clinical trials may begin next year into a gene replacement therapy to replace the disease's faulty gene with a modified, healthy gene.
CF sufferers would inhale the healthy gene which - it is hoped - will replace the faulty gene that causes thick mucus to develop in the lungs.
While this will not stop people carrying the faulty gene, it will disarm the UK's most common life-threatening inherited disease.
For people like Carol Law, of Os-croft, whose twins Kate and Robert were born with Cystic Fibrosis, this is an exciting time.
After years of treatment, Kate tragically died aged 16 from complications after a heart and lung transplant. Robert, now 28, still has daily medication and physiotherapy.
Carol, a regional senior fundraising manager for the Cystic Fibrosis Trust, says successful therapy would increase life quality and expectancy for CF sufferers. She said: 'Anyone with mild to moderate lung damage will benefit from this research because it will stop the progression.'
The Cystic Fibrosis Trust needs about £3m a year to continue this programme of research, and to celebrate Cystic Fibrosis Awareness Week the Big Bounce 2006 has been launched.
Carol said: 'Exercise is vital for CF sufferers, particularly bouncing. Kate often came back blue from tram-polining lessons because she wasn't getting enough oxygen but she knew she had to do it.
It will take until at least 2013 for the treatment to be made widely available.
Carol said: 'The name of the game now is to keep these kids healthy enough to benefit from the gene therapy - it's a race against time and money. The facilities are there, it's just a question of raising enough money to do it.' nIf you are interested in holding a Big Bounce event to raise money for Cystic Fibrosis, contact Bev Burnham-Jones on 0845 859 1029 or see www.cftrust.org.uk
Niall has physio twice every day
SIX-YEAR-OLD Niall Watterson looks like any other boy of his age as he goes to football and Beavers meetings.
But as a Cystic Fibrosis sufferer, Niall has twice-daily physiotherapy, takes medication before eating and is expected to live only to the age of 40.
Broughton Infants School pupil Niall was diagnosed with CF at just eight weeks old, after a routine blood test.
His parents Rachel and Andrew then had to accept that as carriers of the CF gene they had given Niall the disease.
Rachel says: 'Sometimes Niall asks us why do I have to have CF when nobody else does. There's not much we can say except that mummy and daddy gave it to you.'
Rachel added: 'I was quite relieved with his life expectancy when they told us it was 40, because compared to other sufferers it isn't that bad.
'I think if they hadn't diagnosed him so young his life expectancy could have been much less.'
Rachel explained that Niall benefited from a pilot scheme in Wales to screen newborn babies for CF, which is not yet operating in England.
'He was born in the Countess of Chester Hospital, but the tests were done here in Wales.
'If we lived in Cheshire they wouldn't have tested for Cystic Fibrosis, so it was lucky.'
Niall has physiotherapy twice a day, when Rachel and Andrew direct him in a number of squeezing, shaking, blowing, huffing and puffing and coughing exercises and back patting.
Says Rachel: 'It is not painful for Niall but he doesn't like sitting still for so long so it is sometimes difficult.
'We try not to be overprotective, but it is always at the back of your mind, especially when there are coughs and colds going round.'
Niall's bravery resulted in him going to Disneyland Paris with Miles of Smiles children's charity last year.
He also won a competition to have his sixth birthday party hosted by Hariboy, the face of Haribo confectioners.
CF affects vital organs in the body, especially the lungs and digestive system, clogging them with sticky mucus, which makes it difficult to breathe and digest food. CF affects over 7,500 people in the UK.
50% are under 15 and 70% are under 20. In the UK, 2.3 million people carry the faulty CF gene - 1 in 25 of the population.
If both parents are carriers of the faulty gene, there is a 1 in 4 chance with every pregnancy that their child will have CF.