Matt Trudeau, President of ITF Therapeutics, discusses the importance of patient listening in the development of Duvyzat (givinostat) for Duchenne muscular dystrophy (DMD).
DMD is a rare genetic, neuromuscular condition characterized by progressive muscle wasting. Symptoms include progressive weakness and atrophy of both skeletal and heart muscle. Early signs may include delayed ability to sit, stand, or walk and difficulties learning to speak. DMD is caused by genetic changes in the DMD gene.
Just ahead of the second anniversary of its commercial launch, ITF Therapeutics’ givinostat was prescribed to its 2,000th patient in the US. Givinostat is a histone deacetylase (HDAC) inhibitor approved for patients ages six and older with DMD.
Mr. Trudeau highlights the importance of earning trust in the rare disease community, something he explains starts with listening to patients and patient advocates. From attending patient conferences to hiring patient advocacy roles and meeting with advocacy organizations, ITF Therapeutics strives to keep patients at the forefront of their treatment development process.
Improving communication with patients, developing effective educational tools, and removing non-clinical barriers while improving accessibility are other initiatives that Mr. Trudeau deems crucial.
While there is still work to be done in DMD, other indications currently in the pipeline for givinostat include Becker muscular dystrophy and polycythemia vera.
For more information on givinostat, click here.
To learn more about DMD and other rare musculoskeletal conditions, visit https://checkrare.com/diseases/musculoskeletal-diseases/
Importance of Patient Listening in The Development of Rare Disease Treatments