Potentially Protective Agent Shows Benefit in Niemann-Pick Type C
Patients with Niemann-Pick disease type C who received N-acetyl-L-leucine (NALL), an agent that potentially ameliorates lysosomal and metabolic dysfunction, showed reduced neurologic signs and symptoms after 3 months, a phase III crossover trial found. After 12 weeks of treatment, the mean change from baseline in scores on the Scale for the Assessment and Rating of […]
One Class of Antidiabetic Linked With Lower Kidney Stone Risk
One class of glucose-lowering agent may help lower the risk of kidney stones in adults with type 2 diabetes, a cohort study suggested. Adults who newly started on an SGLT2 inhibitor had a 31% lower risk of nephrolithiasis compared with those starting on a GLP-1 receptor agonist (HR 0.69, 95% CI 0.67-0.72), found Julie M. […]
Novel Gout Drug Aces Mid-Stage Tests
SAN DIEGO — An investigational once-daily oral agent that inhibits urate transporter-1 (URAT1) as a means of forcing uric acid excretion showed good efficacy and safety in a pair of phase II trials reported here. In one of these studies, the new product, called AR882 for the time being, reduced serum urate by 40% to […]
Potential Cure for Sickle Cell Clears Hurdle for Approval
An FDA advisory committee meeting on Tuesday did nothing to dispel expectations that the investigational agent exagamglogene autotemcel (exa-cel) will become the first approved gene therapy for sickle cell disease (SCD). Members of the Cellular, Tissue, and Gene Therapies Advisory Committee were not asked to vote on the agent’s efficacy or safety, but instead directed […]
Statin Reduced Risk of Heart Disease in People With HIV
BRISBANE, Australia — The cholesterol-lowering agent pitavastatin (Livalo) as primary prevention reduced the risk of major adverse cardiovascular events (MACE) among people living with HIV, the randomized REPRIEVE trial showed. Over a median follow-up of about 5 years, incidence of any MACE was 35% lower in the pitavastatin group compared with a placebo group, at […]
Agent Orange Exposure Tied to Bladder Cancer Risk in Veterans
Exposure to Agent Orange was associated with a modestly increased risk of bladder cancer among Vietnam War veterans, according to a retrospective cohort study. Among over 2.5 million male veterans, exposure to Agent Orange was associated with a relative 4% higher risk of bladder cancer (HR 1.04, 95% CI 1.02-1.06, P<0.001), reported Stephen Williams, MD, […]
‘Game Changer’ in NASH: Resmetirom Hits Endpoints in Pivotal Study
VIENNA — The investigational agent resmetirom resolved liver biopsy findings in nonalcoholic steatohepatitis (NASH) more often than placebo, according to the phase III MAESTRO-NASH trial. While 10% of placebo patients achieved resolution of inflammation, ballooning, and disease activity, the rate was 26% among people treated with the 80-mg dose of resmetirom and 30% with the […]
Novel PET Imaging Agent Accurately IDs Clear Cell Renal Cancers
An investigational PET imaging agent — zirconium-89 (89Zr)-deferoxamine (DFO)-girentuximab — identified clear cell renal cell carcinoma (ccRCC) with high sensitivity and specificity, according to results from the phase III ZIRCON trial. Use of the agent achieved sensitivity and specificity rates of 85.5% (95% CI 79.8-89.8) and 87% (95% CI 78.8-92.3), respectively, reported Peter Mulders, MD, […]
FDA Moves to Pull Controversial Preterm Birth Drug From the Market
The FDA is calling for withdrawal of the approval for the injectable agent 17α-hydroxyprogesterone caproate (17-OHPC; Makena) for pregnant women with a history of at least one spontaneous preterm birth, stating that the drug is ineffective in reducing preterm births, as well as other poor neonatal outcomes, according to briefing documents released ahead of a […]
First-Line HIV Treatment Proves Mettle in Pregnant Patients
The HIV agent dolutegravir (Tivicay) was tied to greater viral suppression for pregnant patients with HIV-1, according to a cohort study. Among patients who used dolutegravir during pregnancy, viral suppression at delivery was achieved in nearly 96.7% of participants, reported Kunjal Patel, DSc, of the Harvard T.H. Chan School of Public Health in Boston, and […]