Author: Andi Thornton
Biogen update on European access to Spinraza
Here is an update on reimbursed access to Spinraza treatment in the Europe and Middle East region.
Risdiplam update from Roche
On 5th July Roche sent us the following information, as per our request to be kept up to date on their current research regarding the RG7916 development programme which will now be known as “risdiplam”. The programme has now reached a number of key milestones. They also announced a new study called RAINBOWFISH which will…
Spinraza appraisal at NICE
TreatSMA attended an important meeting on Spinraza appraisal at NICE offices in Manchester on 27 June.
Exciting news about new oral molecule for SMA
Risdiplam – a new name for RG7916 – is showing fantastic results in babies participating in the Firefish clinical trial. Almost all the participating babies are progressing well and gaining motor function.
Scotland: new approval pathway for ultra-orphan medicines
People in Scotland with rare diseases may be able to access new treatments in a more efficient manner following the introduction of a new definition of ‘ultra-orphan medicines’.
Encouraging results of a new muscle drug for SMA
Cytokinetics announced results of a recently completed clinical trial of reldesemtiv in SMA. The data suggest that this new experimental drug for SMA increases endurance and respiratory function even over a short period of 8 weeks.
The SUNFISH trial will not be conducted in the UK
TreatSMA has learned that UK will not take part in the SUNFISH trial
SUNFISH trial progressing fast but not in the UK
Recruitment for the SUNFISH clinical trial is about to be completed globally. As of last week, enrollment of participants aged 6–11 and 18–25 years has been completed. But the UK red tape has blocked the sites from opening on time.