The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
Biogen has announced that it will conduct a new global clinical trial, DEVOTE. The study will evaluate if higher dosing of nusinersen can provide improved efficacy in the treatment of SMA across a broad population of patients. The study aims to further build on the long-term safety profile and efficacy of nusinersen in a projected […]
Following a request from SMA Europe, Biogen has released the following community updates on: Access to Spinraza™ : 28 European countries now have access to Spinraza™ for 5q SMA Types I, II and III, via regular reimbursement but in some cases, including age restrictions. Additionally, in certain countries there are rare disease/ medical committees who […]
Cytokinetics announced that the European Medicines Agency (EMA) has granted orphan medicinal product designation to reldesemtiv for the potential treatment of SMA. Reldesemtiv is a drug that has been shown, in the laboratory, to increase the force generated by skeletal muscle and which delays the onset and extent of muscle fatigue. This could be beneficial to people living with SMA […]
It was announced today that AveXis received FDA approval for Zolgensma®, the first gene therapy for paediatric patients with SMA. Zolgensma has been approved in the United States for the treatment of patients younger than 2 years of age who have been diagnosed with SMA and including those who were pre-symptomatic at diagnosis. This treatment […]
Novartis has sent the SMA community a letter announcing that enrollment for the ongoing clinical trial of branaplan is now closed. Branaplam is currently being assessed for safety and efficacy in a Phase 1/2 clinical study in Type 1 infantile-onset SMA. Novartis is pleased to announce that they have enrolled 25 infants into Part 2 […]