The EnAble Fund for Elected Office
04 April 2019 / Posted in: Information, Support
This fund is intended to cover the additional financial costs associated with a disability, that would otherwise prevent someone from seeking elected office.
Your Views On Access To Rare Disease Medicines
04 April 2019 / Posted in: Treatments & Research
Genetic Alliance UK are working on a policy project to improve access to medicines for rare diseases, called Resetting the Model. They have launched a survey to understand your views on how decisions should be made around access to rare disease medicines.
New Rare Barometer Voices Survey on Rare Disease Patients’ Experience of Treatments
27 March 2019 / Posted in: Treatments & Research, Support
This global survey on rare disease patients’ experience of treatments is open to anyone from any country in the world who is living with a rare disease, as well as their family members and carers. It closes on 30th April.
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.
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.
