A gene therapy approved for treating spinal muscular atrophy in infants has also shown improved efficacy in older children.
Roche has presented newest data on safety and efficacy of risdiplam, the next drug being developed to treat all forms of spinal muscular atrophy. The data reinforces earlier observations that risdiplam appears to be safe and effective across the full spectrum of the disease.
The company also announced the dates of regulatory submission in the US and EU.
Friday the 4th of October will be remembered for a very long time. It was the day that ended 2 and a 1/2 years of campaigning and fighting for access to Spinraza for Sam.
We at TreatSMA would love to thank everyone for participating in Saturdays LIVE SMA DAY. We had a whole array of amazing speakers , sports professionals, worldwide known doctors, professionals in SMA and not forgetting those from the community itself. We also had a few visits from the legendary Geordie sausage!
TreatSMA were fortunate enough to be invited to the event along with having a stand for people to come and talk to us about our journey fighting for treatments for rare diseases.
TreatSMA are going live all day on Facebook on the 28th September with a huge event jampacked with speakers covering lots of different aspects of SMA, ranging from physiotherapy to science.
In a recent update we mentioned that hospitals were going to be asked to submit expressions of interest to provide Spinraza for adult patients. We can now confirm that that request has gone out and it is now down to hospitals to submit to NHS England.
Recently there has been a drive from various stakeholders involved in how new medicines or medical devices gain access to the UK market to introduce changes that reflects modern needs of the country. This drive for change generated discussions and…