Good news for patients with rare diseases comes from two studies presented at the American Academy of Neurology congress. In the first, early treatment with Ocrelizumab in relapsing multiple sclerosis (SMR) has been shown to reduce the risk of using walking aids by almost half. The second concerns children with type 1 spinal muscular atrophy (SMA), and has shown that Ridisplam therapy improves survival and motor development.
In the first case, a post-hoc analysis, conducted after 6 years of open-label phase III extension studies, showed that treatment with Ocrelizumab reduced the risk of having to use a walking aid by 49%. patients with relapsing multiple sclerosis (SMR) compared to patients who made the therapeutic switch from interferon beta-1 to two years later.
"For people with MS, keeping mobility as long as possible is extremely important. These new longer-term analyzes are encouraging," said Levi Garraway, Chief Medical Officer and Head of Global Product Development at Roche, the company. who developed the drugs - slowing the progression of MS to an earlier pathological stage, and not just treating relapses, could generate other clinically significant outcomes in people with this disease. "
Part 2 of the Firefish study, presented at the congress, reached the primary endpoint demonstrating a significant improvement in achieving the milestones of motor development in children aged 1 to 7 months after 12 months of treatment. "These results confirm the clinically significant efficacy of risdiplam in children with an advanced and difficult to treat disease - explains Garraway - we thank the SMA community for its collaboration and especially the 62 families around the world who participated in Parts 1 and 2 of the Firefish study ".
In collaboration with:
ROCHE