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, …
Care of patients with Lennox-Gastaut syndrome (LGS) changes substantially when patients reach adulthood and transfer to adult care. Seizure types and frequencies often change with age, behavioral issues may become more difficult to manage, and the introduction of long-term care …
The transition from pediatric to adult care for patients with Lennox-Gastaut syndrome (LGS) is often hindered by fragmented planning and a lack of personalized support. As young adult patients move from home-based care to group homes, assisted living, or long-term …
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 …
A major hurdle in managing type 2 diabetes (T2D) is a widespread gap in clinician awareness around the powerful, far-reaching benefits of GLP-1 receptor agonists (GLP-1 RAs). These innovative therapies help reduce the burden of cardiovascular, kidney, and metabolic (CKM) …
Clinicians face considerable hurdles when diagnosing and managing narcolepsy and idiopathic hypersomnia (IH), largely due to overlapping symptoms, insufficient formal training in sleep medicine, and limited recognition of associated psychiatric and cardiometabolic comorbidities. Compounding the challenge, some therapeutic agents—most notably …
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 …
