The safety and efficacy profile of Ferriprox in patients with thalassemia has led to the study of its potential in other groups of transfused patients, e.g., sickle cell disease and myelodysplasia.
Now, our research into mechanisms of action, has led us to explore the role of our compounds in certain chronic neurodegenerative conditions, in the absence of generalized iron overload, but where iron plays a critical role in the pathophysiology, including: neurodegeneration with brain iron accumulation, Friedrich Ataxia, Parkinson's disease, amyotrophic lateral sclerosis, Alzheimer's disease, multiple sclerosis and Huntington's disease. Both deferiprone and our novel hydroxypyridinones exhibit physicochemical characteristics that favor brain and cellular penetration, making them good candidates for investigation into these disabling conditions, where there are currently inadequate therapeutic options.
ApoPharma is currently conducting research into the hematological disorders of thalassemia and sickle cell disease and the neurodegenerative disorder of Parkinson's disease. You can learn more by going to ClinicalTrials.gov or to the links shown below.
You can be one of the FIRST to contribute to research that may lead to improved outcomes of sickle cell disease.
Safety and efficacy of early-start deferiprone treatment in infants and young children newly diagnosed with transfusion-dependent thalassemia