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We are working with Muscular Dystrophy UK (MDUK), the SMA Trust, and the family led campaign group TreatSMA:

The SMA Type 1 Expanded Access Programme (EAP)

The interim NHS England policy (4th August 2017) will fund the hospital costs of administering this programme until NICE makes a decision on NHS funding. However it has limited which children with SMA Type 1 may have access, to those diagnosed by 7 months of age and with two SMN2 copies. We have joined with clinicians and the other charities and advocacy groups to appeal these limits. You can read more here.

We are very aware that it is not uncommon for a family to experience delays in diagnosis, how distressing this is and how unfair this limit on access would be for any family in this position.

MDUK has a training programme on SMA for GPs. We also try to raise awareness of SMA within the health community. We need to push for more to be done so that children are diagnosed in a timely way. We have asked families to send us their personal stories of delayed diagnosis with permission to forward them to NHS England as a first step and will be working further on this in the next few months.

Long term access for those with SMA Types 1, 2 or 3

Together we have lobbied hard for a ministerial referral to NICE. We expect to hear this month what path the appraisal of the drug for provision in the UK will take. The likely path is as a Highly Specialised Technology (HST). We are concerned that the new HST criteria introduced on 1st April 2017 threaten the possibility of nusinersen eventually being funded by the NHS in England and the long-term potential of this new treatment being available for those affected by SMA (for more information, see ‘Drug Funding and Approvals Process - changes to HST criteria’).

As soon as we know ‘what next?’ with NICE, we will post our plans on this page.