News

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

  1. Update on the clinical development of risdiplam

    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 […]

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  2. EMA validates the Marketing Authorisation Application (MAA) for risdiplam for the proposed use in people living with SMA

    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 […]

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  3. Risdiplam approved for the treatment of SMA in the U.S.

    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 […]

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