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…
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…
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, adolescents and adults with SMA types 2 and 3 who have participated in the SUNFISH trial.
We are thrilled!
AveXis intrathecal trials halted after safety findings
The US Food and Drug Administration has suspended all clinical trials of AVXS-101 (Zolgensma®) gene therapy administered intrathecally due to safety findings in animals.
First Physio with Marion session a great success – catch up here
TreatSMA hosted its first Physio with Marion session on Sunday, and thanks to Marion and all of those who took part in asking questions, it was a great success. You can catch up on a recording of the event above.
Physio with Marion – This Sunday
On the 27th October at 7pm TreatSMA is hosting a live webinar alongside physio Marion Main. This will be a regular slot at the end of each month. It can be viewed on all our social media platforms live and…