As a part of our engagement with research into diet we came across this novel work and thought that general SMA population would be interested in the data.
Our Trustee Gennadiy Ilyashenko has been accepted as a patient organisation representative on the Spinraza managed access oversight committee. This is fantastic news.
Impressive! The results of NURTURE, a presymptomatic clinical trial of Spinraza, are out. The drug appears to prevent SMA altogether in the majority of children when started early!
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.