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Risdiplam update from Roche

  • 6 July 2018

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…

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Spinraza appraisal at NICE

  • 30 June 2018

As everyone knows, TreatSMA along with other patient advocacy groups attended and contributed to an important meeting for Spinraza appraisal at NICE HQ in Manchester on the 27th June.

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Exciting news about new oral molecule for SMA

  • 20 June 2018

On 16th June,  PTC Therapeutics, Inc. presented new interim data from part 1 of the Firefish clinical trial which is investigating risdiplam in babies with type 1 SMA. The compound is showing some fantastic results, with part 2 of the study still ongoing. This is a very exciting time for SMA research.

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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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