Spinraza appraisal at NICE
TreatSMA attended an important meeting on Spinraza appraisal at NICE offices in Manchester on 27 June.
Category: Updates
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.
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’.
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.
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.