UK campaign for access to treatments and support for those with spinal muscular atrophy
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…
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…
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…
TreatSMA had a meeting today with SMA Support UK and The SMA Trust due to the current meetings being held by EMA and their decision expected to be made very soon.
Biogen have provided the following update to the SMA Support UK and SMA Trust in response to their queries:
In infants with spinal muscular atrophy (SMA), treatment with Spinraza (nusinersen), the first FDA-approved drug for SMA, reduces the risk of death or permanent ventilation, according to new results from the Phase 3 ENDEAR study (NCT02193074).
The evaluation of medicines in the EU can be through single national submissions (in the first instance then a mutual review lead by the country who made the first assessment) but in the case of medicines of high importance like neurodegenerative disorders, paediatric medicines and those for rare disease it is normal that the medicines…
I honestly do not know where to start. Finley has totally enriched our lives but inevitably when a diagnosis like this is made, with it there comes pain and mortifying revelation. Our world changed dramatically when he was diagnosed with Spinal Muscular Atrophy Type 2 aged 13 months old in June 2014. This is a…
We were lucky with diagnosis, we’re looked after by a muscular dystrophy centre of excellence. Our team is very positive, we spoke about the need of future ventilation the importance of physio. We were told that it was an exciting time for SMA. Although this was great and we usually have a super positive outlook…
