AveXis Data Reinforce Effectiveness of Zolgensma™ in Treating Spinal Muscular Atrophy (SMA) Type 1
17 April 2019 / Posted in: Treatments & Research
Interim data from Phase 3 clinical trial of Zolgensma (STR1VE) suggest that infants on the gene therapy product live longer and achieve significant developmental milestones.
SMA Europe Reports on its Activities
16 April 2019 / Posted in: Treatments & Research
SMA UK is a member of SMA Europe which held its three-day AGM in Bucharest on 4th-6th April. There were reports on SMA Europe's achievements in the year to date, new organisations were welcomed and current and forthcoming plans were outlined.
Information and Support over Easter
10 April 2019 / Posted in: Information, Support
We are sorry we are unable to stay open for phone or email enquiries over the Easter bank holiday. Find out where you can find information about SMA and support during this time.
DCP - Survey about the impact of caring for a disabled child
10 April 2019 / Posted in: Information, Support
Disabled Children's Partnership are looking to hear from those with parental responsibility for disabled children and young people (from birth to the age of 25) to complete their survey about the impact that caring for their child has had on them and their families. Closes 28th April.
Long-Term Assessment of Salbutamol in SMA Type 3
10 April 2019 / Posted in: Treatments & Research
This latest article from our Clinical Care Research Correspondent, Dr Alex Murphy, discusses how Tiziano and colleagues studied individuals with SMA taking salbutamol over a one year period.
Non-Muscle Related Effects of SMA
09 April 2019 / Posted in: Treatments & Research
Read this article by our Clinical Care Research Correspondent, Dr Alex Murphy, about research by Lipnick and colleagues who used a novel way to look at some of the non-muscle related effects of SMA.
Survey: Accessing Cervical Screening (Smear Test) with a Physical Disability
04 April 2019 / Posted in: Information, Support
Disability Rights UK and Jo's Cervical Cancer Trust are running this survey to find out about experiences in accessing cervical screening (smear test), as well as what provisions are available to enable accessing screening.
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.
