Positive Interim Results from Phase I Trial Announced by Scholar Rock
18 March 2019 / Posted in: Treatments & Research
Scholar Rock have announced positive interim results from an ongoing Phase I trial in healthy volunteers of their lead compound, SRK-015.
Neuro Numbers: The Neurological Alliance Report
14 March 2019 / Posted in: Information, Support
This new report highlights data including that more than one in six people in England are living with one or more neurological conditions, including SMA. It's being used to call for greater prioritisation of neurology by the health and care system.
DCP Looking for Families To Be Part of Secret Life Of Us Media Campaign
13 March 2019 / Posted in: Information, Support
As part of the Secret Life of Us campaign, the Disabled Children’s Partnership are looking for families of disabled children who would be willing to speak, as part of a media campaign, about the impact on daily life and the need for financial and other support.
Study to Explore the Quality of Care Received by Children and Young People on Long-Term Ventilation
11 March 2019 / Posted in: Information, Support
This study is currently being conducted by NCEPOD about the quality of care received by those aged 0-24 years on long-term ventilation (including non-invasive). They are looking to hear from both patients and parent carers for their views.
Neurological Alliance Patient Experience Survey
11 March 2019 / Posted in: Information, Support
The Neurological Alliance is running this survey to find out about the care and treatment for neurological conditions (this includes SMA). The purpose is to provide information which can help them monitor and improve the future quality of health services and social care for people with neurological conditions.
Rare Diseases: Open Letter to the UK Government
08 March 2019 / Posted in: Treatments & Research
Over 125 patient organisations, including SMA UK, have signed an open letter calling on the UK Government to review and refresh, as a matter of urgency, the UK Strategy for Rare Diseases before the end of 2020.
Family Attitudes to Genome Editing for Prevention of Heritable Disorders
08 March 2019 / Posted in: Treatments & Research
This study involves an online survey for people aged 18 or over who either have a genetic condition themselves, or have someone in their family who has / had a genetic condition. It's being led by University College London with a closing date of 30th May.
The Financial and Work-Related Impact of Living with SMA Type 1, 2 or 3 for Unpaid Carers
05 March 2019 / Posted in: Treatments & Research
Thank you to all who responded to this survey, and to Wickenstones for their analysis. The results and the results of our survey last year about the impact of SMA are now available and are being used to inform Biogen’s continuing work with NICE re: access to nusinersen. Read the results summary here.
Letter Written By Clinicians Highlights Frustrations Around Spinraza Access
04 March 2019 / Posted in: Treatments & Research
The letter, published in The Guardian, highlights the clinicians’ frustrations at Spinraza not being made available on the NHS in England, Wales and Northern Ireland.
