Author: Andi Thornton
EAP in the UK
EAP started/starting Newcastle Belfast East Dundee Great Ormond Street Oxford Evelina Leeds Liverpool Glasgow Bristol Cardiff Sheffield (August) Working towards EAP Cambridge Birmingham Oswestry Manchester We’re still waiting on responses from St George’s (London) Contacts we have Evelina Childern’s Hospital: Dr Elizabeth Wraige, elizabeth.wraige@gstt.nhs.uk Cardiff: Dr Louise Hartley, louise.hartley@wales.nhs.uk Newcastle: Prof Volker Straub, volker.straub@newcastle.ac.uk Liverpool: Dr…
EMA opinion on broad-label approval for Spinraza
Committee for Medicinal Products for Human Use of the European Medicines Agency adopts a positive opinion on Spinraza.
Paris option for nusinersen
Dear Members of SMA Community, If any of you want to know more about Paris option for type 1 and be up to date with other developments, please get in touch with TreatSMA members by email on treatsma@treatsma.uk. We are working very hard to help you ALL and you must understand it is not just…
Biogen announces EAP for nusinersen will not be extended to types 2 and 3
Biogen announced that the Expanded Access Programme for nusinersen will not be extended to SMA types 2 and 3.
TreatSMA press release
Every month, across the UK, parents are hearing the devastating words from doctors that their baby is extremely unlikely to survive to see their 2nd birthday. That they will helplessly be forced to watch as their baby grows weaker before their eyes, losing their ability to move their legs; their arms; to lift their head; to…
Position statement from the North Star / SMA-REACH UK Group
On 5 April 2017, the NORTH STAR / SMA-REACH UK group of seventy clinicians, physiotherapists, clinical trial coordinators, industry and advocacy groups’ representatives, including SMA Support UK, sent an open letter, signed on behalf of the group by Professor Francesco Muntoni, setting out their position and recommendations to the National Institute for Health and Care Excellence…