Author: Andi Thornton
Update on adult access to Spinraza
During an election period, Government Departments are restricted in the announcements that they are able to make or the information that they are able to publish. The ongoing communication between NHS England and the Patient Advocacy Groups (PAGs) about implementation of the Managed Access Agreement (MAA) has recently been focused on identification of hospitals with…
Roche updates us on risdiplam timeline
Roche has sent us a statement on risdiplam approval timeline.
Physio with Marion Episode 2
TreatSMA hosted its second Physio with Marion session on Sunday, and thanks to Marion and all of those who took part in asking questions, it was another great success.
FDA grants priority review to risdiplam
The US Food and Drug Administration has accepted Roche’s regulatory filing for risdiplam as a treatment for SMA types 1, 2 and 3
Possibility of an Innovative Drug Fund to be introduced?
Earlier this year TreatSMA met with a number of MPs, including deputy speaker of the House of Commons, to talk about the possibility of introducing a high cost medicine fund.
SRK-015 increases muscle strength in people with SMA
Progress is being made to develop a muscle-strengthening drug in SMA. Scholar Rock, Inc. has just announced interim results of a phase-2 clinical trial of their experimental drug SRK-015. SRK-015 aims to improve muscle strength in people with spinal muscular atrophy by allowing growth of muscle tissue. The drug is a selective myostatin inhibitor –…
Survey about what progress there has been with children’s access to nusinersen in the UK
Over 90 days have passed since NICE published their final guidance for nusinersen meaning that treatment should now be available. TreatSMA, SMA UK and MDUK will all be representing the SMA Community as part of the Oversight Committee for the Managed Access Agreement. The first meeting is not until next year but we remain active.…
Risdiplam is safe and effective in treating SMA type 2 and 3
The world may soon have a new, highly potent treatment for spinal muscular atrophy as Roche today announced that the key clinical trial of risdiplam has met its primary endpoint. Based on the trial data, which are also supported by TreatSMA’s own observations, risdiplam appears safe and highly effective in improving muscle function of children,…