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. Novartis announced that enrollment for the ongoing clinical trial of branaplan is now closed

    enrollment for the ongoing clinical trial of branaplan is now closed

    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 of the study. This is in addition to the 7 (out of 13 infants) who continue to receive branaplam in Part 1 of the study – exposure to treatment ranges from 3 to 4 years. Enrollment to the study is now closed.

    The data from this phase 1/2 study will inform the options Novartis considers for future studies. Novartis is grateful for the dedication of the investigators and their teams who are conducting this study, caring for the enrolled infants and gathering the data.

    In addition, Novartis would like to express its genuine gratitude to all the parents who have considered participation in this study for their children. They truly appreciate the commitment they have made. Novartis would also like to thank the SMA community for its advice and support that has helped them achieve this important stage in branaplam’s development programme.

    Novartis remains committed to its ongoing open communication with the SMA community, as well as to progressing science-based innovation to improve outcomes for people living with SMA. They look forward to continuing dialogue with us – not only to keep us updated on progress with branaplam, but also to gain our insights and feedback on how they can improve their clinical studies and outcomes for SMA patients.

    Please read Novartis’ letter here