Are current advances in gene editing, new drug therapies and less restrictive BMT criteria being explained and rolled out to the sickle cell community in an effective and timely manner? When can people living with sickle cell disease experience a better quality of life on more permanent based on the treatments we already have?
What is needed to translate our knowledge of cardiac development and congenital heart disease genetics into novel diagnostic and/or therapeutic strategies for congenital or acquired heart disease?
Most Pharmacological interventions in cardiovascular diseases tend to address to be used in palliative manner and chronically. There is urgent need for treatments that are curative. This goes to identifying molecular drivers of the diseases and targeting them to correct the underlying pathophysiology rather than symptom suppression.