The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
The European Medicines Agency (EMA) has validated the Marketing Authorisation Application (MAA) for risdiplam for the proposed use in people living with SMA. Validation of the MAA confirms that the submission is accepted and begins the formal scientific evaluation process by EMA’s Committee for Medicinal Products for Human Use (CHMP). Following our request, Roche provided […]
Following our request to receive updates about the risdiplam clinical development programme, Roche is delighted to share with us that the U.S. Food and Drug Administration (FDA) has approved risdiplam for the treatment of SMA in adults and children 2 months of age and older. In the U.S., Risdiplam’s brand name is Evrysdi™. The FDA […]
Following our request, we are pleased to share information regarding a new trial Biogen is planning to initiate, the RESPOND trial. Despite the significant advances made in recent years, there are still unmet needs and opportunities to improve care for infants and children with SMA. Available data in the long-term study of Zolgensma (onasemnogene abeparvovec-xioi) […]
Based on our request for an update, Biogen has provided us with the following information about new results from their NURTURE study, a study of pre-symptomatic patients with SMA, recently presented at the virtual Cure SMA Research & Clinical Care Meeting, which took place from 10th to 12th June 2020. NURTURE is an ongoing, Phase […]
Roche presented new data on Part 1 of the SUNFISH trial, as well as on the JEWELFISH study, at the virtual Cure SMA Annual Conference, which took place between 8th and 12th June. SUNFISH SUNFISH is a large (231 participants) global two-part study in children and adults, aged between 2 and 25 years, with Type […]