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 ...

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 ...

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 ...

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 ...

The risk of occurrence of the clinical trial's endpoints was lower in patients with PPF treated with 9 mg or 18 mg twice a day compared with those given the placebo. Treatment with nerandomilast at a ...

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 ...

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 ...

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 ...

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 ...

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 ...

Benefits of selumetinib extended to sleep, fatigue, mobility, and social and occupational functioning. Adults with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas ...

Abby Bronson advocates for those with Becker muscular dystrophy, which can have severe consequences on mobility and quality of life. Larry Luxner, senior correspondent for Rare Disease Advisor, ...