04 December 2018 / Posted in: Treatments & Research
Pharmaceutical company Scholar Rock has announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has adopted a positive opinion on their experimental drug for SMA, SRK-015.
29 November 2018 / Posted in: Information, Support
Newlife are calling on the government to help those children whose lives are at risk because they don’t have the specialist equipment they need. Their report, 'Equipment Crisis for Disabled Children', outlines a four-point-plan to improve equipment services for disabled children and their families.
29 November 2018 / Posted in: Treatments & Research
As an act of ‘good faith’ in their commitment to progress discussions to a satisfactory conclusion, we urge all three parties to agree with immediate effect to implement, a ‘bridging solution’ for access to nusinersen for any infants with SMA Type 1.
28 November 2018 / Posted in: Information, Support
On Wednesday 21st November, we joined MDUK at Parliament for the launch of this inquiry, which included discussing ways to improve the availability of appropriate psychological support. You can read their full APPG report here.
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.
26 November 2018 / Posted in: Information, Support
Able Move, a start-up company that has created a portable solution for disabled travellers, has been chosen as winner of this award, in partnership with Leonard Cheshire. The company was founded by Josh Wintersgill, a cyber-security manager from Bristol, who has SMA.
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: Information, Support
A consultation being run by the Office of Rail and Road (ORR) sets out proposals to change the Disabled People's Protection Policy (DPPP) Guidance for train and station operators on how to write their policies for helping disabled people to travel by rail.
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.