Novartis announces 25 infants with SMA Type 1 now enrolled into Part 2 of their trial

20 May 2019

Novartis Community Update – 25 infants with SMA Type 1 now enrolled into Part 2 of  their trial.

This means enrolment for this trial has now closed. 

Branaplam is currently being assessed for safety and efficacy in a Phase 1/2 clinical study in Type 1 infantile-onset SMA. These 25 infants are in addition to the 7 (out of 13 infants) who continue to receive branaplam in Part 1 of the study. Their exposure to treatment ranges from 3 to 4 years.

The data from this phase 1/2 study will inform the options Novartis considers for future studies.

In a letter to SMA Europe, Novartis express their thanks to: the investigators and their teams who are conducting this study, caring for the enrolled infants and gathering the data; to all the parents who have considered participation in this study for their children; to the SMA community for its advice and support.  

You can read Novartis' letter here