Recent advances in the treatment of hemophilia, a genetically X-linked bleeding disorder, include non-factor prophylactic therapies such as factor VIII mimetics, anti-tissue factor pathway inhibitors (anti-TFPIs) and small interfering RNA (siRNA) treatments. Health care professionals (HCPs) treating patients with hemophilia …
Recent advances in the treatment of hemophilia, a genetically X-linked bleeding disorder, include non-factor prophylactic therapies such as factor VIII mimetics, anti-tissue factor pathway inhibitors (anti-TFPIs) and small interfering RNA (siRNA) treatments. Health care professionals (HCPs) treating patients with hemophilia …
Immunoglobulin A nephropathy (IgAN) is a progressive glomerular disease and a leading cause of chronic kidney disease (CKD), with up to 40% of patients advancing to kidney failure within two decades. Despite guideline-recommended management emphasizing supportive care and RAAS inhibition, …
Generalized myasthenia gravis (gMG) is a chronic neuromuscular disorder that results from destruction of the neuromuscular junction by pathogenic autoantibodies, partially through the actions of the neonatal Fc receptor (FcRn). Traditional therapies only partially alleviate the heavy disease burden and …
Suboptimal health care and poor health care outcomes for individuals with intellectual and developmental disabilities (IDDs) often stem from health care professionals (HCPs) lacking necessary skills to treat these individuals. It is crucial that HCPs receive education on how to …
Transthyretin amyloidosis (ATTR), including hereditary variants such as ATTRv-CM and ATTRv-PN, remains underdiagnosed despite increased awareness and treatment advances. Many patients experience significant diagnostic delays, often seeing multiple clinicians before receiving a correct diagnosis, which can limit treatment efficacy and …
