Among 360 patients with the progressive genetic disorder transthyretin amyloidosis (or ATTR amyloidosis), 12 months of treatment with the RNA interference therapeutic agent patisiran “resulted
Genetics can help screen for patients’ potential for subsequent malignant neoplasms (SMNs), researchers report. “A polygenic risk screening approach may be a valuable complement to
Patients with disabling pansclerotic morphea (DPM) caused by gain-of-function variants in the gene for signal transducer and activator of transcription (STAT) 4 (STAT4) responded to ruxolitinib therapy, a
For the 89,000 patients with cystic fibrosis worldwide, mucolytics, anti-inflammatories, and antibiotics should be used for first-line pulmonary treatments, authors of a review article conclude,
The small-molecule compound inaxaplin (VX-147) reduced proteinuria in a small phase 2a study of patients with 2 apolipoprotein L1 (APOL1) variants, biopsy-proven focal segmental glomerulosclerosis, and
As recommended in product labeling, liver enzyme monitoring is needed during the first 18 months of tolvaptan therapy and every 3 months thereafter, according to
In a Mendelian randomization (MR) study, associations were identified between genetic variants associated with type 2 diabetes and increased risk of several gastrointestinal diseases (GD),
FDA last week approved velmanase alfa (Lamzede), the first enzyme replacement therapy approved in the U.S. for the treatment of non-central nervous system manifestations of alpha-mannosidosis,
A 12-gene pharmacogenetic panel significantly reduced the incidence of clinically relevant adverse drug reactions when implemented across diverse European healthcare systems, researchers report. “Large-scale implementation”
Efanesoctocog alfa — an agent that provides high sustained factor VIII activity by overcoming the von Willebrand factor–imposed half-life ceiling — provided clear clinical benefits