Treating teenagers and adults with oral risdiplam, a potential therapy for spinal muscular atrophy (SMA), led to sustained increases in blood levels of the key SMN protein missing in these patients, the lead investigator for a Phase 2 clinical trial said in an interview.
Jake Ogborne and TreatSMA visited the Houses of Parliament to meet with Jacob Rees-Mogg to highlight his and others’ need for treatment.
The core team at TreatSMA, met on Saturday 13th July to discuss ongoing activities into accessing treatment for SMA and also to discuss our future plans.
Jake Oborne and family show the emotional impact being ineligible for treatment has on SMA sufferers. Click below to watch this powerful video.
Following the miscommunication from NICE in terms of releasing information TreatSMA, Spinal Muscular Atrophy UK and Muscular Dystrophy UK contacted NICE to express our unhappiness with the communications sent out. We have now received a response which you can read below.
TreatSMA are asking for those not eligible for Spinraza, to contact MPs and get involved in the debate.
Come and join us for a night of fun, family, community and celebration.
Now that the new MAA has been released we wanted to share with you the process that we have gone through.