The simple act of breathing can be a daily struggle for adults living with Duchenne.
NHS England & NICE have recently announced that the process for approval of new drugs is about to change. These proposals – set to be implemented on 1st April – will impose significant constraints on the way that new drugs like Raxone are assessed and funded in England.
During my early 20s, I had multiple chest infections, these episodes affected my lung function so I required overnight ventilation. Growing accustomed to overnight ventilation was frustrating because of pressure sores from wearing the ventilator mask, finding the machine too noisy to fall asleep and also changing positions would mean having to adjust the mask.
Part of living with Duchenne means having to accept using the ventilator in the daytime which can be tough to use in public as it cannot be hidden or disguised. I lost confidence in myself as I didn’t recognise the person in the mirror.
My voice is quiet & I can easily get short of breath without the ventilator so I use it when have to speak for long periods. This dilemma is the toughest to overcome as there is no alternative to using respiratory support. At the time I wished there was a magic pill or medication to improve respiratory function. Initially, I restricted myself from leaving the house whilst using my ventilator but that made me feel angry, frustrated and isolated. I realised I had to change my perspective regarding ventilation. I could not shut myself away any longer because I am an outgoing person who enjoys socialising and leading a purposeful life.
I’m extremely interested in any drug such as Raxone that can improve or prolong respiratory function with some relevance concerning adults living with Duchenne. I strongly feel that the worst part of living with Duchenne is having breathing difficulties and restricted communication.
I urge NHS England to review the guidelines as these new proposals will adversely affect patient access to treatments for rare diseases. This is a shame as research into DMD treatments is moving extremely fast since the approval of Translarna.