The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
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 […]
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) […]