The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
We are delighted to share with you that the Committee for Medicinal Products for Human Use (CHMP), of the European Medicines Agency (EMA), adopted a positive opinion recommending the approval of risdiplam for the treatment of 5q SMA, in people 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type […]
In response to our request, Biogen shared an update on the RESPOND trial with us. The RESPOND trial is a Phase 4 open-label, multicentre study which evaluates the efficacy and safety of nusinersen in patients with a suboptimal clinical response to gene therapy (onasemnogene abeparvovec). It was announced today that the first patient has been […]
Roche has provided us with a summary of its work in the community throughout this year, including information about the risdiplam clinical development programme and progress towards their goal of making risdiplam available to all patients who can benefit from the treatment. Ensuring patient safety, uninterrupted access to treatment and to support the wider community: […]
Scholar Rock reported on interim analysis results from their TOPAZ clinical trial to evaluate the safety and efficacy of SRK-015 in people with SMA Types 2 and 3. SRK-015 is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by SMA patients. The drug is an antibody that very specifically targets […]
Following our request to receive important and timely information about the risdiplam clinical development programme, Roche has shared with us an update on part 1 of the FIREFISH clinical trial. FIREFISH is an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants with the […]