19 September 2017 / Posted in: Research
Roche have provided an update on clinical trials of their SMN2 splice-modifying drug RG7916
30 August 2017 / Posted in: Research
You can now read the full workshop report written by the clinical researchers and patient representatives from Europe and the US who met last year.
29 August 2017 / Posted in: Information & Support
Are you a young disabled person living in London and looking for employment support or an organisation looking to broaden your employment opportunities? Find out more about MDUK's employability project here.
24 August 2017 / Posted in: Research
Sent on 23rd August from clinicians and advocacy groups, this asks for the age limit and SMN2 copy number restrictions on potential access to be removed.
21 August 2017 / Posted in: Information & Support
Genetic Alliance UK is now running a survey to understand patients' experiences including any challenges they face, or concerns they have, when applying for insurance. This information will help them to update the resources available on their website.
16 August 2017 / Posted in: Research
Roche has begun to enrol SMA patients in the FIREFISH Phase II trial of their splice-modifying drug, RG7916 (also known as RO7034067).
16 August 2017 / Posted in: Information & Support
The Disabled Children's Partnership recognise what holidays are like for disabled children and young people, and their families. Here, they list some of their top tips.
07 August 2017 / Posted in: Research
We have clarified that, from now on going forwards (until we hear from NICE), children will have to be diagnosed by 7 months of age to be eligible. If you have a child who has already been diagnosed with SMA Type 1 who is older than this and not already on the EAP, please read more.
04 August 2017 / Posted in: Research
All hospital costs for administering nusinersen to patients with SMA Type 1 will be provided by NHS England.
02 August 2017 / Posted in: Information & Support
Contact a Family are concerned that around 100,000 parents with disabled children are at risk of being worse off on Universal Credit. Sign their petition to the prime minister to reverse cuts to child disability payments under Universal Credit.