Tel: 01789 267520

Roche’s SUNFISH study community update

19 September 2017 / Posted in: Research

Roche have provided an update on clinical trials of their SMN2 splice-modifying drug RG7916

Read full story

Revisiting the Consensus Statement for Standards of Care in SMA - Full Report

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.

Read full story

Muscular Dystrophy UK's 'Moving Up' Employability Project

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.

Read full story

Letter to NHS England Requesting Review of EAP Type 1 Interim Policy Criteria

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.

Read full story

Help Genetic Alliance UK Produce the Insurance Information you Need

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.

Read full story

RG7916 FIREFISH trial now enrolling patients (though not in UK)

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).

Read full story

The Disabled Children's Partnership: Top Tips for the Summer Holidays

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.

Read full story

Age of children who will be able to access the EAP for SMA Type 1 going forwards

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.

Read full story

NHS England Announces Interim Policy on EAP for SMA Type 1

04 August 2017 / Posted in: Research

All hospital costs for administering nusinersen to patients with SMA Type 1 will be provided by NHS England.

Read full story

New Campaign: Tell the PM to Stop Universal Credit Cuts

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.

Read full story