This is an encouraging time with many clinical trials in development, and several treatments either available or emerging on the horizon. Once treatments are available it is crucial that people are able to access them quickly. When access to treatments is delayed it is families who suffer. Our FastTrack to treatments campaign is committed to identifying and removing the barriers that cause delays to ensure that patients are able to access and benefit from new, safe and effective treatments as quickly as possible.
Support for the campaign
Professor Dame Kay Davies, Dr Lee’s Professor of Anatomy at the University of Oxford:
Muscular Dystrophy UK continue to make significant contributions to muscle disease research. Through their Fast Track strategy they now plan to make a difference in the delivery of the fruits of this research to the clinic. This is a vital step in getting novel treatments for patients. I am delighted to hear that Muscular Dystrophy UK continues to be at forefront in this challenging area.
Professor George Dickson, Chair of Molecular Cell Biology at Royal Holloway:
We are in a promising time with new therapies finally coming to the market. It is vital that people with muscle-wasting conditions are able to access life-changing new treatments as quickly as possible, this is why I fully support Muscular Dystrophy UK’s new Fast Track campaign.
Dr Adnan Manzur, Consultant Paediatric Neurologist at Great Ormond Street Hospital:
Muscular Dystrophy UK have been at the forefront of bringing novel treatments from clinical trials to the patient. Their work with NICE was instrumental in developing and implementing the Managed Access Agreement which allowed ataluren (Translarna) treatment to be made available to boys with nonsense mutation Duchenne muscular dystrophy. Further advocacy of early access to novel medicines and initiated is like the fast track campaign, are most important.
Professor Francesco Muntoni, Professor in Paediatric Neurology and Director of the Dubowitz Neuromuscular Centre:
The new Fast Track campaign is very timely and welcome. There are currently very few treatment options for many muscle-wasting conditions. When new treatments become available it is crucial that they get to those who need them as quickly as possible. Without these drugs, people’s conditions can decline at least for one condition, SMA1, there are major implications for the survival of affected individuals if drugs are not started in a timely fashion. The focus needs to be on removing barriers and accelerating access to these new treatments, and identify appropriate mechanisms to deal with rare and very rapidly progressive conditions, an area which there does not appear to be a dedicated path in this country.
Mary Glindon MP, Chair of the All Party Parliamentary Group for Muscular Dystrophy:
As the Chair of the All-Party Parliamentary Group for Muscular Dystrophy I strongly welcome the new Fast Track campaign from Muscular Dystrophy UK. Without the right assessment processes and funding in place we risk individuals and families enduring agonising delays before people with muscle-wasting conditions can benefit from cutting-edge therapies, which could drastically improve their quality of life.