Novartis announces 25 infants with SMA Type 1 now enroled into Part 2 of their trial
20 May 2019 / Posted in: Treatments & Research
This means enrolment for this trial has now closed. Branaplam is currently being assessed for safety and efficacy in a Phase 1/2 clinical study in Type 1 infantile-onset SMA. These 25 infants are in addition to the 7 (out of 13 infants) who continue to receive branaplam in Part 1 of the study. Their exposure to treatment ranges from 3 to 4 years.
NICE Announces Guidance on Nusinersen will be Published on 26th June
15 May 2019 / Posted in: Treatments & Research
NICE have now updated their website with this advice. This means that until 26th June, Biogen will fund currently untreated infants with SMA Type 1 via a “bridging solution”. All patients already on the EAP will continue to have access to treatment. Those with SMA Types 2 and 3 will then start to get access subject to the terms of the MAA and as soon as services are able to cope with the patient load.
NHS England Announces Access to Nusinersen for all with SMA Types 1, 2, 3a and 3b
15 May 2019 / Posted in: Treatments & Research
The treatment will be made available by Biogen immediately to the youngest and most severely-affected infants (SMA Type 1). The NHS will then begin to provide nusinersen to those with SMA Types 2 and 3 shortly after NICE’s full guidance is published, and once the services to deliver them are established. It will also be available to pre-symptomatic siblings who, based on their genetics, are very likely to develop SMA.
Mental Health Awareness Week 2019
14 May 2019 / Posted in: Information, Support
This week is Mental Health Awareness Week. Read Rare Disease UK's report from 2018 about mental health and the impact of living with a rare disease, and find out more about how we may be able to help with emotional support.
Potential Biomarker for SMA Identified
10 May 2019 / Posted in: Treatments & Research
Biogen presented data at the American Academy of Neurology 2019 conference on a potential biomarker for SMA.
Pre-symptomatic Infants Treated with Spinraza™ Achieve Motor Milestones More Consistent With Normal Development
10 May 2019 / Posted in: Treatments & Research
Data from the NURTURE study showed that pre-symptomatic treatment of infants very likely to develop SMA Type 1 or 2 due to their inherited genes resulted in motor milestone achievements more consistent with normal motor development.
AveXis Presents Positive Interim Results on Zolgensma Across a Broad Spectrum of Patients with SMA
07 May 2019 / Posted in: Treatments & Research
AveXis presented positive results from clinical trials on Zolgensma, across a broad spectrum of patients with SMA at the 2019 American Academy of Neurology conference in Philadelphia.
Data Published in Neurology Suggest Long-Term Benefits of Spinraza™ Treatment in Later-Onset SMA
07 May 2019 / Posted in: Treatments & Research
Biogen has announced that data from a study on the safety and tolerability of Spinraza™ in individuals with later-onset SMA had been published in the peer-reviewed journal, Neurology.
Education Secretary Seeking Views On Funding For Children With Special Needs
03 May 2019 / Posted in: Information, Support
Damian Hinds is at a conference of the National Association of Head Teachers today where he is expected to make an announcement calling for a fresh look at educational funding for children with special needs in England.
Response From Biogen to SMA UK & MDUKs' Letter
03 May 2019 / Posted in: Treatments & Research
Following SMA UK and MDUK sending letters urging Biogen, NHS England and NICE to work strenuously this week so that the NICE committee can endorse an agreement for access to nusinersen for all with SMA Types 1, 2 & 3, we have received a response from Biogen.