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A potential side effect added to Spinraza label

  • 30 July 2018

The European label of Spinraza will soon list a new potential side effect: hydrocephalus. We don’t yet know whether this severe condition, which was experienced by only five patients treated with Spinraza, is at all linked to nusinersen, the active ingredient of Spinraza. In response to the Community’s queries Biogen has provided the following update:

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Innowalk – Parent Review

  • 23 July 2018

Please note this is not an official Innowalk post, this is a review compiled by an owner of an innowalk who has no affiliation to the company and the views are their own * As the website will state “The…

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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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