Mental Health Awareness Week 2019
14 May 2019 / Posted in: Information, Support
This week is Mental Health Awareness Week. Read Rare Disease UK's report from 2018 about mental health and the impact of living with a rare disease, and find out more about how we may be able to help with emotional support.
Potential Biomarker for SMA Identified
10 May 2019 / Posted in: Treatments & Research
Biogen presented data at the American Academy of Neurology 2019 conference on a potential biomarker for SMA.
Pre-symptomatic Infants Treated with Spinraza™ Achieve Motor Milestones More Consistent With Normal Development
10 May 2019 / Posted in: Treatments & Research
Data from the NURTURE study showed that pre-symptomatic treatment of infants very likely to develop SMA Type 1 or 2 due to their inherited genes resulted in motor milestone achievements more consistent with normal motor development.
AveXis Presents Positive Interim Results on Zolgensma Across a Broad Spectrum of Patients with SMA
07 May 2019 / Posted in: Treatments & Research
AveXis presented positive results from clinical trials on Zolgensma, across a broad spectrum of patients with SMA at the 2019 American Academy of Neurology conference in Philadelphia.
Data Published in Neurology Suggest Long-Term Benefits of Spinraza™ Treatment in Later-Onset SMA
07 May 2019 / Posted in: Treatments & Research
Biogen has announced that data from a study on the safety and tolerability of Spinraza™ in individuals with later-onset SMA had been published in the peer-reviewed journal, Neurology.
Education Secretary Seeking Views On Funding For Children With Special Needs
03 May 2019 / Posted in: Information, Support
Damian Hinds is at a conference of the National Association of Head Teachers today where he is expected to make an announcement calling for a fresh look at educational funding for children with special needs in England.
Response From Biogen to SMA UK & MDUKs' Letter
03 May 2019 / Posted in: Treatments & Research
Following SMA UK and MDUK sending letters urging Biogen, NHS England and NICE to work strenuously this week so that the NICE committee can endorse an agreement for access to nusinersen for all with SMA Types 1, 2 & 3, we have received a response from Biogen.
SMA UK and MDUK write to NICE, NHS England and Biogen
02 May 2019 / Posted in: Treatments & Research
Read the flyer and letters that were sent urging these three organisations to work strenuously this week so that on May 8th, the NICE committee can endorse an agreement for access to nusinersen for all with SMA Types 1, 2 and 3.
Urgent Call For Action - Please Contact Your MP Today About The NICE Committee Meeting on 8th May
01 May 2019 / Posted in: Treatments & Research
Following yesterday's announcement to say access to nusinersen is now to be considered at a private fourth NICE committee meeting, we need to pull out all the stops and ask our MPs to write to all parties urging them to reach an agreement on access for all.
Update from NICE re Nusinersen
30 April 2019 / Posted in: Treatments & Research
Update from NICE re nusinersen: The outcome of discussions, following the meeting on 6th March, and the consequences for access to nusinersen, will be considered by the committee on Wednesday 8th May.
