We currently have limited information but we wanted to get news out to you as soon as possible. The MHRA have approved the use of risdiplam for type 1 and type 2 patients over the age of two months with no upper age limit.
George and Lucy were featured in this week’s Sunday Mail, one of the largest newspapers and websites in the world reminding everybody about the benefits of treatment and that TreatSMA continues to publicise and fight for treatments to be available.
In this webinar we were joined by the charity SpecialEffect who specialise in providing assistive technology to those who are unable to use standard gaming controllers and so forth to help get them back into the world of computer gaming.
In this webinar we had the pleasure of being joined by Dr Giovanni Baranello who is a senior Clinical Lecturer in Neurosciences at Great Ormond Street Hospital as well as a Clinical Lecturer in Paediatric Neurology and Neuromuscular Disorders at…
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.