Results of the phase II-III trial of AMO-02 in congenital DM1

The results of the international REACH-CDM trial involving 56 children aged between 6 and 16 with the congenital form of myotonic dystrophy type 1 were announced in a press release on 6 September 2023.

• The primary endpoint, the Clinician-Completed Congenital DM1 Rating Scale, was not met.
• A significant reduction in CPKEnzyme contenue dans les cellules musculaires, qui est libérée dans le sang en cas d’atteinte musculaire. enzymes was measured in children treated with AMO-02, as well as better performance in the 10-minute walk test for functional capacity and in the Peabody picture vocabulary test for cognitive capacity.

AMO Pharma announces affirming data from REACH-CDM clinical trial for AMO-02 in treatment of myotonic dystrophy AMO Pharma, 6 September 2023