In 2020, the US Food and Drug Administration approved 53 novel drugs. Thirty-six of the 53 (68%) drugs were reviewed and approved through an expedited review pathway, and 31 of the 53 (58%) were approved for treatment of a rare disease. This review includes a summary of the novel drugs approved by the US Food and Drug Administration in 2020.Recent years have seen important advances in our understanding of calciphylaxis, especially regarding newly identified risk factors and histologic findings that may aid diagnosis. This retrospective study of cases of calciphylaxis treated in our hospital in the last 13 years focuses on newly revealed aspects of this disease. We describe 16 patients (62.5% women; mean age, 67.9 years). In addition to advanced kidney disease (in 75% of our patients), other factors associated with the presence of calciphylaxis were a history of treatments related to phosphorus and calcium metabolism (75%) and anticoagulation (62.5%), usually with vitamin-K antagonists. Histology showed alterations in elastic fibers in only 25% of the biopsy specimens. Eleven of the patients died sepsis was most often the cause.

To examine provision of direct-to-patient medication abortion during COVID-19 by United States family physicians through a clinician-supported, asynchronous online service, Aid Access.

We analyzed data from United States residents in New Jersey, New York, and Washington who requested medication abortion from 3 family physicians using the online service from Aid Access between April and November 2020. This study seeks to examine individual characteristics, motivations, and geographic locations of patients receiving abortion care through the Aid Access platform.

Over 7 months, three family physicians using the Aid Access platform provided medication abortion care to 534 residents of New Jersey, New York, and Washington. There were no demographic differences between patients seeking care in these states. A high percentage (85%) were less than 7 weeks gestation at the time of their request for care. The reasons patients chose Aid Access for abortion services were similar regardless of state residence. The maphic access to medication abortion.

We measured women’s preferences for avoiding an unintended pregnancy. We determined if young age (<25) was associated with lowest utility with an unintended pregnancy.

We conducted a cross sectional study of women presenting for hormonal contraception who did not desire a pregnancy. We used four techniques to elicit health prefences and calculate utilities for an unintended pregnancy visual analog scale, and willingness to pay, time-tradeoff (TTO), and standard gamble. We dichotomized each measure to define lowest utility for each measure. We used predicted probabilities and multivariable logistic regression to estimate the association between age (≤25 vs ≥26 y) and lowest utility with an unintended pregnancy.

Our sample included 419 participants from four states. Selleck Tanespimycin We found that younger age (≤25) was positively associated with reporting the lowest utility for unintended pregnancy. In absolute terms, with the visual analog scale, the probability that a woman 25 years or younger would have lower preference for an unintended pregnancy was 26.8% (95% CI 20.4-33.2%) versus (21.7% (95% CI 14.3-29.0%). Using the willingness to pay, the probability of the younger group having lower preference was 84.9% (CI 80.3-89.4%) compared to 57.3% (CI 49.3-65.3). With the TTO, Women 25 years old and younger had a 78.3% probability (CI 72.6-84.0%) of low utility on the TTO vs 48.9% (CI 40.9-56.9%) in the older group. With standard gamble, younger women had a 47.0% probability (CI 36.8-50.6%) versus 18.0% (CI 14.7-27.5%).

Women of all ages report a decrease in health utility with unintended pregnancy. This decrease in health utility is greater among young women (age <25).

Health utilities for unintended pregnancy can be used to guide cost effectiveness research and health policy.

Health utilities for unintended pregnancy can be used to guide cost effectiveness research and health policy.Lipoprotein-proteoglycan binding is an early key event in atherosclerotic lesion formation and thus conceivably could play a major role in vasculopathy-driven chronic graft failure and cardiovascular mortality in renal transplant recipients. The present study investigated whether lipoprotein-proteoglycan binding susceptibility (LPBS) of apoB-containing lipoproteins and levels of the classical atherosclerosis biomarker LDL-C were associated with cardiovascular mortality (n = 130) and graft failure (n = 73) in 589 renal transplant recipients who were followed up from at least 1 year after transplantation for 9.5 years. At baseline, LPBS was significantly higher in patients who subsequently developed graft failure than in those with a surviving graft (1.68 ± 0.93 vs. 1.46 ± 0.49 nmol/mmol, P = 0.001). Cox regression analysis showed an association between LPBS and chronic graft failure in an age- and sex-adjusted model (hazard ratio 1.45; 95% CI, 1.14-1.85; P = 0.002), but no association was observed with cardiovascular mortality. LDL-C levels were not associated with graft failure or cardiovascular mortality. This study shows that measurement of cholesterol retention outperformed the traditionally used quantitative parameter of LDL-C levels in predicting graft failure, suggesting a higher relevance of proatherogenic function than the quantity of apoB-containing lipoproteins in chronic kidney graft failure.The serine palmitoyltransferase (SPT) complex catalyzes the rate-limiting step in the de novo biosynthesis of ceramides, the precursors of sphingolipids. The mammalian ORMDL isoforms (ORMDL1-3) are negative regulators of SPT. However, the roles of individual ORMDL isoforms are unclear. Using siRNA against individual ORMDLs, only single siORMDL3 had modest effects on dihydroceramide and ceramide levels, whereas downregulation of all three ORMDLs induced more pronounced increases. With the CRISPR/Cas9-based genome-editing strategy, we established stable single ORMDL3 KO (ORMDL3-KO) and ORMDL1/2/3 triple-KO (ORMDL-TKO) cell lines to further understand the roles of ORMDL proteins in sphingolipid biosynthesis. While ORMDL3-KO modestly increased dihydroceramide and ceramide levels, ORMDL-TKO cells had dramatic increases in the accumulation of these sphingolipid precursors. SPT activity was increased only in ORMDL-TKO cells. In addition, ORMDL-TKO but not ORMDL3-KO dramatically increased levels of galactosylceramides, glucosylceramides, and lactosylceramides, the elevated N-acyl chain distributions of which broadly correlated with the increases in ceramide species.