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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The health authorities in Belgium and the Netherlands have announced that Spinraza treatment will be made available in both countries this summer.
Below is an update on reimbursed access to Spinraza treatment in Europe and the Middle East. The news was provided to us today by Biogen in response to our request.
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…
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.
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.
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.