Lobby of Welsh Assembly - Pauline - Stopwasting - Action Duchenne - Fighting for a cure for muscular dystrophy

Lobby of Welsh Assembly
10 June 2009

As advocate for the region I organised the lobby of Welsh Assembly, working with AM Christine Chapman who kindly sponsored the event and Ian Griffiths, a young man living with the condition. Christine is Ian's AM and has met him several times, she was keen to help us in this campaign.

The day before the lobby I received several calls from families and health care professionals alerting me to the fact that the current provision in Wales was to receive another blow. From now on, respiratory services for pediatrics was set to change. All sleep studies are now to be carreid out not in Wales, but at Great Ormond Street or Glasgow! This raised even more concerns about care for those with Duchenne in Wales.

We met outside the Senned, it was great to see the families and children who turned up to voice their concerns and meet with their AMs. 40 family members congregated for pictures, spoke with press and then went in to ensure their AMs received copies of the standards of care consultations and our lobby document.

The event meeting itself was very well attended, with families, specialist newborn screening nurse Rachel Salmon, several AMs and Minister for Health Edwina Hart.

The event meeting was started with a video presentation by Ian Griffiths, who clearly stated the need for a specialised centre of excellence in Wales and the urgent need for better health care provision for those affected by Duchenne. Ian could not attend personally as he does not have the necessary portable equipment to feel safe and comfortable away from his home.
Ian's presentation was well recieved with a loud round of applause afterwards.

I then spoke about the Action Duchenne standards of care survey and consultations, the strengths and weaknesses this revealed, and how we need a set standard of care for all with Duchenne in line with Treat NMD recommendations. Families in Wales are having to wait 2 years for a "one off" appointment at Great Ormond Street, it is apparent that Wales needs a centre of excellence. Families are opting to get their child under the care of a centre of excellence as they see care in Wales as sub-standard, yet these centres in England are obviously already stretched.

Christine Chapman AM then said a few words about her involvement in the campaign and her reasons for supporting us, as she has met Ian several times and wanted to do all she could to help.

Nick Catlin, Action Duchenne CEO then gave a presentation on the effect that care standards for young men with Duchenne has on quality and length of life, with specific reference to care received in Denmark. Young men in Denmark have a much higher life expectancy than here in the UK.
He spoke about perceived quality of life; studies show that health professionals rate this as very poor for young men with Duchenne, yet the young men themselves rate their quality of life as good. Why would they NOT wish to lengthen a good life? Health professionals run the risk of being biased in their level of treatment by their own misconceptions on quality of life of those with Duchenne.
Nick stated the need for a centre of excellence in Wales, that services in the 2 centres in England are already stretched. He spoke of how current treatments already require very careful monitoring by a team of experts, and the need for such teams to be available to all, especially in light of new drugs (PTC 124 and the AVI exon skipping drug) on trial right now, which will provide a "tailor- made" treatment for each individual. The safe administration and monitoring of these drugs has to be considered as a top priority, and so now is the time to act to ensure the facilities and teams are in place for the families in Wales.

Rachel Salmon then spoke of the new born screening service and the lack of any professionals she can refer families on to. With other conditions there would be a consultant and specialist nurse who would then form part of the care team. For those diagnosed with Duchenne there are no such support services. She stated the need for proper information given during ante natal stage, and the urgent need for specialists she can refer families on to for information and treatment plans after diagnosis.

Edwina Hart stood and spoke, assuring everyone that she had taken on board the issues raised and heard the needs stated which would be given her consideration. Nick Catlin urged her to consider placing a centre of excellence in Wales, with the pledge that Action Duchenne would fund the first years wage for a professional who could head this centre. It was stated by Nick Catlin that there would soon be the need to access new drugs currently on trial, if this cannot be done safely in Wales this will mean families travelling to GOSH, which would mean higher costs. It will be economical to set up a center in Wales for families to access trial drugs along with meeting the care needs, rather than have to fund more staff at GOSH and cover travel costs for families having to access GOSH as things stand. Edwina Hart has agreed to a further meeting with us to discuss the issues raised and how to address them.

The signed petition was handed to Edwina Hart by Matthew Barrett, a teenager who has Duchenne, before she left the event.

Questions were raised by families, who expressed anger at the lack of care and equipment in Wales, and dismay that Ian could not attend in person. Christine Chapman was obviously moved by the plight of all affected, admitting she had known little of the needs before the event, and pledging her support to try to rectify the issues we face by working with us, and how she had felt reassured by Edwina Harts words that we could work towards addressing the problems.
A parent then read a letter sent to him by Edwina Hart last year in which she stated the services for respiratory care would be stronger within 12 months. He expressed disbelief at her words today as news had just broken that sleep studies could not even now be undertaken in Wales, it seems the service has actually deteriorated, not become stronger.
I closed the meeting by thanking Christine Chapman who had so kindly sponsored the event, and who was moved to tears by what she had learned. Christine has pledged to work with us and help us in our fight for better care, and has already contacted me to arrange another meeting.