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.
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.
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…
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…
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!
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.