Latest News: Treatments & Research
28 November 2018 / Posted in: Treatments & Research
The UK SMA Patient Registry collects clinical and genetic information from individuals living with SMA to provide an aid to clinical study recruitment and an invaluable resource to the research community. To find out more about the registry and a snapshot of the data held, please click here.
23 November 2018 / Posted in: Treatments & Research
On 15th November, Biogen was announced as the winner of the Orphan Product Award for Spinraza® (nusinersen). They comment, ‘we must see this translated into availability for the patients who desperately need it’. We agree and will keep working on this.
19 November 2018 / Posted in: Treatments & Research
The theme for the day has been announced as ‘Bridging Health and Social Care’. The 12th edition of the campaign will focus on the need to bridge the gaps in the coordination between medical, social and support services for people affected by a rare disease.
13 November 2018 / Posted in: Treatments & Research
Last week, along with MDUK, we were patient reps at NICE’s scoping meeting for AVXS-101 for SMA Type 1. We also attended Genetic Alliance UK’s workshop for rare disease groups discussing a Patient Charter for Newborn Screening in the UK.
12 November 2018 / Posted in: Treatments & Research
This latest SMA Community update from NICE doesn't give a decision yet but states that talks are still progressing.
01 November 2018 / Posted in: Treatments & Research
Despite our urgent request, Biogen has not extended the cut off for this programme to 1st December. It ends now for any newly diagnosed infants. Read their community statement here.
26 October 2018 / Posted in: Treatments & Research
Thank you to the patient and clinical experts who strongly advocated for access to nusinersen at NICE’s committee meeting this week; to clinicians, families and adults who: expressed their views in their submissions to NICE; wrote letters to MPs, NICE, NHS England and Biogen; were there on the day.
19 October 2018 / Posted in: Treatments & Research
AveXis yesterday submitted their regulatory applications for AVXS-101 in the U.S., Europe and Japan for use in infants with SMA Type 1. This is an important initial step toward the potential approval of AVXS-101, a gene replacement therapy candidate for the treatment of the underlying root cause of SMA.
18 October 2018 / Posted in: Treatments & Research
An article in The Guardian, published yesterday, describes how C Frank Bennett, senior vice president of research at Ionis Pharmaceuticals, and Adrian Krainer of Cold Spring Harbor Laboratory, have won the prize for developing Spinraza.
SMA UK’s submission to NICE’s scoping consultation on AVXS-101 treatment for infants with SMA Type 1
17 October 2018 / Posted in: Treatments & Research
England’s regulatory body, NICE, has invited a wide range of clinical and patient groups to comment on this treatment. You can read what we have said to NICE here.