Research into Duchenne has reached an unprecedented stage, with a tidal wave of emerging treatments now in the pipeline. However, if these are to be developed it is essential the UK has the capacity to host clinical trails, capable of linking patients with innovative emerging drugs and proving the clinical efficacy of said treatments.
Our ‘Joining the Dots’ initiative and partnership in the ‘Newcastle Plan’ therefore aims to develop the capacity of the UK to host clinical trials and ensure every person living with Duchenne in the UK has access to clinical research opportunities and the highest standards of multidisciplinary care.
In the summer of 2014, Translarna (Ataluren) became the first European Medicines Agency approved treatment to address the underlying causes of Duchenne muscular dystrophy. Since this date we have campaigned tirelessly to ensure this treatment receives funding approval and is made available to all those eligible to benefit.
We are also fighting to ensure that the UK has a quick, robust, transparent and fair process for evaluating future Duchenne treatments, and to guarantee the mistakes, misguidance and inertia displayed in NICE and NHS England in their evaluation of Translarna, cannot be repeated. These efforts extend to focussing upon the regulatory framework of clinical trial and drug approval, encouraging funding for Duchenne research and the development of improved and adaptive regulatory pathways.
Despite huge advances in research and the clinical management of Duchenne, a collective failure to acknowledge the potential of every child living with Duchenne has led to many living unfulfilled lives in adulthood.
In addition raising to expectations amongst patients and families, we further inform health, education and social care professionals about the learning and behavioural difficulties associated with Duchenne, and the need for good educational outcomes as a basis for a well rounded adult life.