- In June 2024, Avidity Biosciences launches Phase III “HARBOR” trial for del‑desiran (AOC 1001) Avidity advanced its siRNA therapeutic targeting the mutated DMPK RNA into a larger Phase III study, following strong Phase II results showing improved muscle strength and reduced myotonia in early-phase trials
- In March 2024, Dyne Therapeutics announces positive interim results from Phase II “ACHIEVE” trial of DYNE‑101 The optimized antisense oligonucleotide showed safety and promising reductions in myotonic symptoms and fatigue scores, prompting continuation of development
- In August 2021, AMO Pharma focused on developing treatments for several rare neuromuscular disorders, including Myotonia Congenita. The company actively engaged in clinical trials to assess the efficacy of its investigational drugs in enhancing muscle function and alleviating symptom severity in patients affected by Myotonia Congenita. This commitment underscores AMO Pharma's dedication to addressing the unique challenges faced by individuals with this condition through innovative therapeutic solutions
- In December 2022, AMO Pharma Limited announced that it had successfully completed patient enrollment for its REACH-CDM study, which is focused on the investigational therapy AMO-02 for treating congenital myotonic dystrophy. This milestone signifies a crucial step in advancing the clinical evaluation of AMO-02, aimed at addressing unmet medical needs in this patient population. The completion of enrollment highlights AMO Pharma's commitment to developing innovative therapies for congenital myotonic dystrophy
