For PBC, the phase 3 GLISTEN trial of linerixibat showed a significant pruritus improvement, with an adjusted mean difference of –0.72 on the Worst Itch Numeric Rating Scale. Ileal bile acid ...
In all, 218 proteins showed a significant association with a cardiac index. Four correlated with both LVEF and Ecc, and 42 with both LVEF and LGE. None correlated with all 3 indices. Researchers ...
Comparison with a concurrent cohort of 47 patients with clinically diagnosed wild-type ATTR-CM revealed a striking difference in disease burden. Systematic age- and biopsy-guided screening during ...
The relative left ventricular wall thickness, indicating relative wall thickening, was significantly higher in patients who were technetium-99m pyrophosphate (99mTc-PYP) positive compared with ...
Exploratory outcomes indicated further benefits, including lower rates of myasthenic crises and hospitalizations, as well as reduced need for rescue therapy. Cemdisiran, a small interfering RNA (siRNA ...
Several genes previously associated with loss of ambulation showed no evidence of interaction with corticosteroid exposure. Genetic modifiers, particularly those in the transforming growth factor β ...
Benefits of selumetinib extended to sleep, fatigue, mobility, and social and occupational functioning. Adults with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas ...
Investigators will enroll approximately 100 boys with early ambulatory stage DMD in a new 72-week randomized controlled trial. Delandistrogene moxeparvovec (Elevidys ™), the first approved gene ...
Oral corticosteroids were the most common treatment used by patients with systemic autoimmune rheumatic disease-associated interstitial lung disease whose disease had progressed to PPF. A recent study ...
The Arg185 residue of nucleotide-binding oligomerization domain-like receptor pyrin domain-containing protein 12 (NLRP12) is the direct binding site for obeticholic acid, and this interaction reduces ...
New information regarding neonatal physiology related to massive hemorrhage, treatment approaches, and the current data can assist in informing evidence-based recommendations regarding the management ...
Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results