Prophylactic cardiac treatment in DMD patients is associated with a 54% lower hazard of death, highlighting its potential survival benefit. Only 27.7% of DMD patients received prophylactic cardiac ...
Despite the availability of long-term prophylaxis (LTP), a substantial proportion of patients with hereditary angioedema (HAE) in Sweden continue to require repeated on-demand or short-term treatments ...
Garadacimab-gxii is the first FDA-approved factor XIIa inhibitor for hereditary angioedema, offering a once-monthly prophylactic treatment option. The VANGUARD trial showed an 87% reduction in HAE ...
Matched cohort studies revealed infrequent adverse events (AEs) with either treatment, but the number needed to harm for any AE was more than three times higher with colchicine vs NSAIDs. The ...
Rare diseases pose a substantial burden to patients and the healthcare system, comparable to those of cancer and heart failure. [i] Thankfully, advances in diagnosis and healthcare delivery can help ...
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