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Celebrating Rare Disease Day 2019

  • 17 February 2019

In less than two weeks we will start celebrating Rare Disease Day – that annual day when all those who are affected by a rare disorder go out and speak about it. The global rare disease community holds celebrations throughout the world. This year TreatSMA will take part with an online campaign and a face-to-face event in the South East.

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First presymptomatic baby receives gene therapy in London

  • 7 February 2019

Clinical development of gene therapy for SMA made a huge leap forward last Tuesday when doctors at the Great Ormond Hospital in London successfully administered AVXS-101 in a new clinical study. A newborn girl became the first child in the UK to receive the ground-breaking treatment presymptomatically.

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NICE will review Spinraza again on 6 March

  • 1 February 2019

For the third time a drug appraisal committee of the National Institute for Health and Care Excellence will meet to review the nusinersen (Spinraza) dossier. The meeting will take place on the 6 March in Manchester.

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FAQ asked about type 1 SMA patients

  • 23 January 2019

Below are some the most frequent questions and problems that we are seeing within the Type 1 community. We have compiled a guide based on the evidence from the newly Published Standards of Care, AA diet website and other documents or protocols that are used within the community.

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