We need your help
JTSMA Chair Max Huxham has been recruited as a patient group representative onto the TREAT NMD Steering Committee for UK Clinical trials. This is for the proposed clinical trial with the new European TROPHOS drug specifically designed for Types II & III SMA (see http://www.trophos.com/ )
It would be really useful to be able to collect some of your views on the general concept of new drug development for SMA.
So if you would like to help you can simply send us an e-mail answering these three questions.
- How do you rate the importance of clinical trials for SMA - very important / fairly important / not important / no opinion
- What do you consider to be the most important use for a therapeutic drug in SMA - for example; improvement in function, strength, range of movement, prevention of further deterioration, walking, breathing etc. please explain your thoughts.
- What is your opinion on the use of drugs which reduce the effects of SMA but do not provide a cure?
If you have any other thoughts or opinions on the matter please feel free to include those as well. All e-mails will be dealt with in the strictest confidence and no names will be used in any resulting documentation
Please e-mail your answers to
By letting us know your thoughts we help the clinicians and scientists better understand how to give the best Help for Today and Hope for Tomorrow
Thank you.
Max Huxham
Chair of Trustees
and
Chris Reed
Director of Support Services | 
