Accelerating and Improving the Diagnosis of Wilson Disease
Untreated Wilson disease can eventually result in death, but patients who receive adequate treatment can have a normal lifespan
Untreated Wilson disease can eventually result in death, but patients who receive adequate treatment can have a normal lifespan
Dr. Rahul Banerjee of the University of Washington discusses the benefits and drawbacks of using synthetic control arms in randomized controlled trials.
L-glutamine was approved in 2017 to treat acute complications in patients with sickle cell disease, but uptake of and adherence to it have been limited.
Given the rarity of CAD and lack of comparative trials, treatment plans are primarily based on findings from nonrandomized trials and clinical experience.
Researchers worked to better understand and manage patients with mild or moderate hemophilia, who can be an underrepresented and underserved population.