Author: Marni Smyth
St George’s Hospital, London is now treating Adults with SMA who are eligible under the MAA for Spinraza
We are happy to update that St George’s Hospital in London is now treating Adults with SMA who are eligible under the managed access agreement with Nusinersen. If anyone in their area would like more information please see website (https://www.stgeorges.nhs.uk/service/neuro/neurology/neuromuscular-disorders/spinal-muscular-atrophy-service)
Survey exploring access to Standards of Care for people with Spinal Muscular Atrophy (SMA)
As part of a PhD project, the John Walton Muscular Dystrophy Research Centre in Newcastle are conducting a survey to explore access to Standards of Care for people with Spinal Muscular Atrophy (SMA). They have asked us to spread the word within the SMA community, they would also like to make it clear that only…
Roche Answers Questions from The Patient Group on the UK Early Access to Medicines Scheme for Risdiplam
This information is provided in response to a series of questions shared jointly with Roche in writing by UK patient organisations Muscular Dystrophy UK (MDUK), TreatSMA and SMA UK. Unless otherwise stated this information applies to people living across the UK.
Trustee Joseph Irwin looks back at 30 years of changes in EU legislation in drug development for rare diseases in his latest paper
TreatSMA trustee Joseph Irwin looks back at 30 years of changes in EU legislation in drug development for rare diseases in his latest paper.
European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for Risdiplam.
European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for risdiplam for the proposed use in patients with spinal muscular atrophy (SMA).
TreatSMA take on their most ambitious SMA Awareness Month to date!
This year, TreatSMA wanted to present a range of topics for the community, but they were also particularly keen to remember and acknowledge those that fought/fight and got the community where it is today, not just in regards to SMA but disability itself. The ongoing battle for equality and disability rights fought by so many should…
Further Clarity on Access to Compassionate Use Program for Risdiplam.
We have received further clarity statement from the NHS England Clinical Panel for nusinersen with regards to access compassionate program for Risdiplam.
Spinraza – Access for Adults FAQs
NICE and NHS have provided FAQs on adults accessing Spinraza.