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’.
UK campaign for access to treatments and support for those with spinal muscular atrophy
BLOG ARCHIVE
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.
The SUNFISH trial will not be conducted in the UK
TreatSMA has learned that UK will not take part in the SUNFISH trial
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.
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.
Registration for NICE meeting on Spinraza now open to public
NICE Appraisal Committee will meet on 27 June to discuss draft recommendation for Spinraza provision in England and Wales. You are welcome join!
Registration for NICE meeting on Spinraza now open to public
NICE Appraisal Committee will meet on 27 June to discuss draft recommendation for Spinraza provision in England and Wales. You are welcome join!