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Scotland: new approval pathway for ultra-orphan medicines

  • 19 June 2018

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’. ‘Ultra-orphan medicines’ will include medicines that can treat people with very rare conditions affecting fewer than 1 in 50,000 people, or approximately 100 individuals in Scotland.

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Encouraging results of a new muscle drug for SMA

  • 17 June 2018

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.

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The SUNFISH trial will not be conducted in the UK

  • 14 June 2018

Yesterday TreatSMA have been made aware that the Great Ormond Street Hospital for Children will NOT be taking part in the Roche SUNFISH trial that was to include SMA patients with types 2 and 3. This means that the trial will not be conducted in the UK at all.

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SUNFISH trial progressing fast but not in the UK

  • 8 June 2018

The SUNFISH clinical trial is progressing fast, and all of its planned 186 participants will likely be recruited in the next few weeks. As of last week, enrollment of participants aged 6–11 and 18–25 years has been completed globally. However, nobody in the UK in those age groups will participate in this long-awaited trial as the all-too-prevalent red tape has impeded the opening of UK trial sites.

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