Blog posts
In this video we got to see Andi attempting to use the Jaco robotic arm. An amazing piece of technology that has been around for some time now and gives those who are unable to use their arms effectively the freedom to be able to pick up items, open doors, pickup glasses of water (and beer of course) as well as a whole host of other things.
TreatSMA trustee Joseph Irwin looks back at 30 years of changes in EU legislation in drug development for rare diseases in his latest paper.
Earlier today we published a series of questions and answers that we got from Roche relating to the recent news about risdiplam, and in particular, the expansion of the compassionate usage programme (CUP). It was a long document but we wanted to get it out to you as quickly as possible. However, we thought it might be useful to give you a quick summary of the key points.
Dr John Bach is a world renowned expert in non-invasive respiratory care. A legend in his field he has well over 600 papers published as well as books that I used across the world to help manage all types of…
Have you ever wondered what worms and SMA have in common? No, me neither, until now. Our very own science geek G hosted a webinar with Dr Maria Dimitriadi to talk about that and many other fascinating topics. Another fascinating…
We were delighted and truly honoured to have Baroness Tanni Grey-Thompson join us as part of SMA Awareness Month to talk about her life, her experiences, discrimination and answer some of your questions. Such a thought-provoking and and inspirational discussion…
We joined a session with Roche earlier this week to ask them questions following their recent announcement about risdiplam. The following FAQs have been checked for accuracy with Roche where they relate specifically to risdiplam.
Questions and answers apply to people living in England, Scotland, Northern Ireland and Wales.
A really interesting webinar hosted by our very own Joseph talking to Dan from the MHRA about the regulatory process associated with rare diseases and their involvement in assessing suitability and helping licensing drugs.