It is an exciting time for research into Myotonic Dystrophy. From ‘in the lab’ to patient trials, there is more happening now than ever before.
CureDM regularly updates a ‘Medication Snapshot’ which you can view and download here.
The headings below will take you to individual research, pharma updates and studies – please do reach out if you have any questions. CureDM are proud to be a connection between patients and pharma/trials.
AMO-02 (tideglusib) is in development for the treatment of congenital myotonic dystrophy and has potential for use in additional CNS, neuromuscular and oncology indications.
PGN-EDODM1 is a novel, potentially disease-modifying, peptide-conjugated oligonucleotide candidate, currently being studied in patients with myotonic dystrophy type 1 (DM1).