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Also known as Sanfilippo syndrome, this rare genetic syndrome has no known cures and what current treatment options are available are expensive and limited in supply.

This year’s most-read articles on autoimmune diseases explored comorbidities with cardiovascular disease, the value of new forms of monitoring and therapy, and more.

A vaccine is showing promising results in treating melanoma; the Biden administration will implement inflation penalties on dozens of drugmakers to reduce out-of-pocket costs for Medicare recipients; the CDC issued an alert urging health care providers to increase immunization coverage for influenza, COVID-19, and respiratory syncytial virus (RSV).

The results indicated that a substantial proportion of patients, particularly in rural, less educated, and elderly demographics, are insufficiently informed about novel MM treatments, including chimeric antigen receptor (CAR) T-cell therapies and bispecific antibody alternatives.

The approval of belzutifan, an oral hypoxia-inducible factor-2 alpha inhibitor, provides a new option for patients with advanced renal cell carcinoma (RCC) following treatment with a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a vascular endothelial growth factor tyrosine kinase inhibitor (VEGF-TKI).

The addition of serplulimab to carboplatin and nab-paclitaxel significantly prolonged survival vs chemotherapy alone when used in the first-line treatment of patients with previously untreated locally advanced or metastatic squamous non–small cell lung cancer.

Trastuzumab administered subcutaneously delivered unique immunomodulation effects vs intravenous trastuzumab in patients with treatment-naïve HER2-positive (HER2+) breast cancer

Coverage of our peer-reviewed research and news reporting in the health care and mainstream press.

Givinostat, a histone deacetylase inhibitor from Italfarmaco, was supported by data from the phase 3 EPIDYS study, a randomized, double-blind, placebo-controlled, multicenter trial investigating the agent vs placebo.

The treatment developed by Amgen could be the first therapeutic option approved for third-line treatment of advanced small cell lung cancer (SCLC).

Skeletal muscle proved to be significant in calculating chemotherapy toxicities in nonmetastatic colorectal cancer (CRC).

A new report from AHIP shows that Medicare Advantage outperformed traditional Medicare on several clinical quality measures, including preventive screenings. However, debate continues over these plans’ cost efficiency and impact on the financial sustainability of rural hospitals.

New research shows that adding navy beans to one’s usual diet may be a viable dietary strategy for modulating the gut microbiome and regulating host markers associated with metabolic obesity and colorectal cancer.

This year’s most-read articles on multiple sclerosis (MS) explored the emergence of new medications, modes of therapy, and predictive tools in the study of MS.

The rate of invasive pneumococcal disease in children plummeted by 72% from 2002 to 2021; health care spending in the United States rose 4.1% in 2022 to $4.5 trillion; roughly two-thirds of Americans who have a mental health condition were unable to access treatment in 2021 despite having health insurance.

While past studies have shown the connection between poor sleep and job performance, this research dives deeper into the experience of this specific population.

Children with sickle cell disease and their caregivers experience an increased burden on health-related quality of life (HRQoL) due to the frequency of vaso-occlusive crises (VOCs).

Improved psychological distress and physical function were among the benefits of doing aerobic exercise and tai chi as a patient with lung cancer.

The US Preventive Services Task Force (USPSTF) recommended that children with obesity receive intensive counseling at age 6 to promote healthy diet and exercise habits; a new survey found that the majority of American patients are wary of their doctors using artificial intelligence (AI); a class-action lawsuit was filed Tuesday against health insurer Humana for using an AI algorithm that systemically denies seniors rehabilitation care recommended by their doctors.

From 2013 to 2022, the all-cause mortality rate among patients with HIV in China declined from 5.4% to 2.7%.

Patients with metastatic colorectal cancer (CRC) who experience colon perforation may have similar survival times to those without perforation.

The commercial market got slightly less competitive while the Medicare Advantage market got a little more competitive, but both markets remain highly concentrated.

BCMA-directed CAR T-cell therapy anitocabtagene autoleucel demonstrated a 76% complete remission rate and 89% negative minimal residual disease in patients with relapsed/refractory multiple myeloma.

Patients with age-related macular degeneration (AMD) who were not responsive to anti–vascular endothelial growth factor agents were able to increase their fluid-free intervals with faricimab.

In AUGMENT-101, patients with R/R KMT2Ar acute leukemia had high overall response rates; despite being heavily pretreated, 40% were able to proceed to transplant and the KMT2Ar cohort was stopped early.

Ciprofloxacin, ceftriaxone, and piperacillin/tazobactam showed the greatest associations with adverse drug reaction case reports related to C difficile.

A late-breaking abstract presented at the American Society of Hematology Annual Meeting and Exposition supports the addition of subcutaneous daratumumab to standard-of-care bortezomib, lenalidomide, and dexamethasone (VRd) in patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplantation.

Investigators explored the genetic relationship between psoriatic disease and serum lipid levels.

Adding metformin to the insulin regimen for pregnant patients with preexisting type 2 diabetes (T2D) or gestational diabetes diagnosed early in pregnancy did not reduce the risk of neonatal adverse outcomes.

End-of-year victory for lawmakers as health care policies pass in the House; insurance limitations prompt concerns over access to semaglutide (Ozempic) for diabetes; a legal battle ensues over the fate of abortion laws in Arizona.