25 May 2017 / Posted in: Research
Cytokinetics has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to its compound CK-2127107 for the potential treatment of SMA.
16 May 2017 / Posted in: Research
Biogen responds to NHS England's requests about the UK EAP for infants with SMA Type 1; they also tell us when they hope to hear from NICE.
11 May 2017 / Posted in: Research
We asked about the position in the UK and overseas.
10 May 2017 / Posted in: Research
You can now read the latest information about NHS England’s position on considering the possibility of supporting the costs of delivering the EAP in England. We now have authority to share information from the meeting we attended on 25th April with the NHS England Specialised Commissioning team.
09 May 2017 / Posted in: Information & Support
Explaining SMA and why it happens is quite difficult. This book is aimed at children aged 8 and over but it can also be read to a younger child, be used in schools or when explaining SMA to friends.
08 May 2017 / Posted in: Help Us
We are revamping our Coffee and Cake Day packs and are looking to feature two recipes. Enter your family favourites now! Closing date is Friday 26th May at 5.00pm.
04 May 2017 / Posted in: Information & Support
This year, Together for Short Lives will be turning up the volume on children’s palliative care to raise awareness. Find out how you can get involved.
03 May 2017 / Posted in: Information & Support
Lidia, Maddison's mum, shares their family's thoughts and experiences in this SMA Voices piece about Maddison who was diagnosed with SMARD at 9 months and is now 8 years old.
27 April 2017 / Posted in: Information & Support
Calling all individuals, families and professionals. Please tell us what you think about current services and give your thoughts on our future direction in our survey. Closing date for responses is 5pm on Friday 26th May 2017.
26 April 2017 / Posted in: Research
Cytokinetics has announced that it has initiated enrolment of a second cohort of SMA patients with SMA Types 2, 3 or 4 in their phase 2 clinical trial of CK-2127107, in North America.