Limited Global Access to Zolgensma announced by Avexis
20 December 2019 / Posted in: Treatments & Research
Clinicians and SMA patient groups in SMA Europe have welcomed the news that Avexis will be launching a global compassionate use programme in January 2020 for eligible children under 2 years of age, but have concerns about how it will operate.
Clinicians & Patient Groups Requesting AveXis Clarification About Potential Limited Global Access to Zolgensma
19 December 2019 / Posted in: Treatments & Research
We're aware that complex and sensitive information regarding potential but very limited global access to the gene therapy treatment Zolgensma has been shared online. Clinicians and patient groups have expressed their concerns to the drug company AveXis requesting, as a matter of urgency, public clarification of the position.
New SMA Voices: Gary Shares His Experiences
18 December 2019 / Posted in: Information, Support
In this video, Gary looks back at his childhood and forward to present day, and shares his experiences of what it’s like to live with Spinal Muscular Atrophy.
Information and Support over the Christmas Break
11 December 2019 / Posted in: Information, Support
Our office is closed from 1pm on Friday 20th December 2019. We re-open on Thursday 2nd January 2020 at 9.00am. Here are links to where you can access support and information about SMA during this time.
Together for Short Lives Launches Youth-Led Creative Artwork Project
10 December 2019 / Posted in: Information, Support
Called 'This Is Me', the project provides an opportunity for young people with life-limiting conditions in the UK to express themselves through the creative arts. Submissions deadline is 20th January 2020.
Support the #NeuroPledge2019
03 December 2019 / Posted in: Information, Support
The Neurological Alliance are asking parliamentary candidates to take the #NeuroPledge2019. It commits them to campaigning for improvements for people with neurological conditions, if they become an MP. Find out how you can get involved and write to your prospective MP.
Nusinersen Managed Access Agreement – Update on Progress with Services for Adults
02 December 2019 / Posted in: Treatments & Research
NHS England has let the Patient Advocacy Groups (PAGs) know which hospitals now have contracts to offer adults treatment, and their plans to address any service gaps.
CONCORD Survey about the Coordination of Care for Rare Conditions
28 November 2019 / Posted in: Information, Support
The COordiNated Care Of Rare Diseases (CONCORD) research study is currently looking for patients, carers and healthcare professionals to share their priorities and views about the coordination of care for rare conditions, via their online survey. Survey closes Sunday 8th December.
FDA Grants Priority Review to Risdiplam for the Treatment of SMA
25 November 2019 / Posted in: Treatments & Research
The FDA’s decision will be announced on 24th May 2020. If approved, risdiplam, an orally administered liquid, would be the first at-home administered medicine for people living with SMA.
Scholar Rock Reports Preliminary Pharmacokinetic and Pharmacodynamic Data from TOPAZ Phase 2 Trial of SRK-015 for the Treatment of SMA
25 November 2019 / Posted in: Treatments & Research
SRK-015 is an experimental therapy directed at the muscle, that aims to reverse or restrict the atrophy and weakness experienced by SMA patients.
