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Researchers identified variants of 9 genes that increase the risk of developing the rare disease.

A comparison of patients with heart failure with preserved ejection fraction (HFpEF) randomized to 1 of 2 physical activities did not show a significant increase in peak oxygen consumption (VO2) vs guideline-based physical activity.

Cutting-edge genetic tools diagnosed 83% of Irish patients with polycystic kidney disease (PKD), identified 36 novel variants, and discovered that seemingly unrelated individuals with the same variant likely inherited it from a common ancestor.

The peptide-drug conjugate rapidly delivers an alkylating payload into tumor cells.

An FDA advisory panel Friday unanimously endorsed the third US COVID-19 vaccine, a 1-shot vaccine produced by Janssen, a subsidiary of Johnson & Johnson (J&J), and an emergency use authorization by the FDA is expected to follow shortly.

Among caregivers of patients with Parkinson disease experiencing psychosis, only 11% learned about the symptom from a physician, with communication challenges attributed to having to prioritize other issues in a limited appointment time.

The study examined various factors used in an internationally developed risk stratification strategy to assess which ones had evidence of being significant in determining a child’s prognosis from the deadly disease.

Patients with osteoarthritis-related pain were shown to benefit from cognitive behavioral therapy for insomnia (CBT-I) via telephone, in which the treatment significantly improved sleep and fatigue after 12 months, with pain temporarily relieved as well.

Compared with adult-onset multiple sclerosis (MS), pediatric-onset MS has few treatments directed at the condition, specifically those that promote remyelination, enhance neuroprotection, and remediate cognitive deficits.

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

Nanotherapies have the potential to overcome the blood-brain barrier and the blood-cerebrospinal fluid barrier, representing a possible treatment opportunity for rare diseases.

More Americans say they are willing to receive a COVID-19 vaccine; 50 million doses have been administered during President Joe Biden's first 5 weeks in office; FDA allows Pfizer vaccine to be stored at standard freezer temperatures.

This week, the top managed care news included an FDA committee meeting on J&J's COVID-19 vaccine; cemiplimab approved for first-line advanced non–small cell lung cancer with PD-L1 expression; real-world outcomes contrast cancer clinical trial results in Medicare patients.

The results also showed that the infants achieved certain milestones in motor development, pulmonary function, and survival.

While only 3 small interfering RNA (siRNA)-based therapies have been approved, the market is poised to expand with 7 other candidates in phase 3 trials.

Break Through Cancer, a collaborative research effort between 5 top US academic cancer centers, aims to pursue solutions to some of the most difficult challenges in cancer research.

A survey found that 42% of US working adults were more stressed or anxious in the past year about taking a sick day to care for themselves or someone else compared with years past, with 1 sick day currently associated with a $227 earnings loss.

Genetically decreased vitamin D levels are unlikely to have a large effect on risk of type 1 diabetes, according to results of a Mendelian randomization study published in PLOS Medicine.

A new survey conducted by Horizon Therapeutics highlighted the negative impacts thyroid eye disease have on patients’ quality of life, finding effects often continue beyond the acute disease phase and into a chronic phase.

Adherence to guidelines for slowing chronic kidney disease is low, with substantial variation among the United States and other countries.

Outsiders take advantage of a Los Angeles program aimed to aid communities hit hard by COVID-19; Biden plans to send 25 million masks out to Americans; real-world data highlight the efficacy of the Pfizer/BioNTech COVID-19 vaccine.

Although the exact underlying pathogenesis of migraine remains unknown, lifestyle modifications with regard to stress, sleep, exercise, and dietary considerations have been reported as beneficial for moderate clinical features.

Among 6 sphingosine-1-phosphate (S1P) receptor modulators evaluated for use in patients with multiple sclerosis (MS), a new meta-analysis shows amiselimod had the highest efficacy.

A recent study found that patients immunized against influenza were less likely to test positive for or have serious complication related to coronavirus disease 2019 (COVID-19).

An FDA committee will discuss emergency use authorization of the coronavirus disease 2019 (COVID-19) vaccine from Johnson & Johnson (J&J); American Cancer Society issues a report on cancer survivor concerns; Tennessee is found to have wasted thousands of vaccine doses.

On this edition of Managed Care Cast, we present a short excerpt from a January webinar titled “Color and COVID-19: The Virus’ Disproportionate Impact” hosted by the MJH Life Sciences™ COVID-19 Coalition about how the pandemic has had an unequal, devastating impact on communities of color.

Pulmonary function increased during sleep for patients with chronic obstructive pulmonary (COPD) receiving a triple-therapy regimen, but improvements to quality of life remain to be seen.

Georgetown investigators attempted to answer how type of supportive relationship may affect outcomes among gay men living with and without HIV, comparing results seen with primary and secondary relationships and having no support.