Gillian McGovern, Associate Editor

The mutations, C594Y and P735R, were found in the GNE gene and may influence sialylation, a process that is crucial for brain development.

Pediatric patients with the variant were approximately 1.44 times more likely develop ALL compared with children who do not have the variant.

The ODD comes after AP303 presented meaningful improvements in renal survival in an ADPKD and the completion of the first study that evaluated healthy human participants.

The manager of clinical pharmacy at UPMC Health Plan describes the significance of the pharmacist’s role in multiple sclerosis care and when collaborating with other health care providers.

The authors note that these findings provide opportunities for health care professionals to utilize social media to reach patients and share accurate information.

The authors believe that pancreatic enzyme therapy has potential in managing patient symptoms, altering the disease course, and the development of standardized care.

The study authors are hopeful that this finding can help in the development of new drugs or treatments for patients with multiple myeloma.

A study that analyzed data from 2000 to 2021 indicated improvements for both leukemia-free survival and overall survival for older patients with acute myeloid leukemia.

Early treatment administration with this regimen also promoted a faster decrease in viral load and shorter viral shedding in patients with hematologic diseases who were positive for COVID-19.

The test scans whole blood samples from individual human donors for the 5 parasite species that can cause malaria in humans and detects Plasmodium RNA and DNA.

The state order was signed on Tuesday and is just a part of an overall mission to strengthen and protect reproductive rights within New York for citizens and those out of state.

The orphan drug designation (ODD) comes after positive results from a phase 2 trial that showed cevidoplenib improved platelet counts by 63.6% and 40.9% in 2 different dose groups.

The indication is for adults and adolescents with moderate to severe immune compromise due to medical conditions or immunosuppressive medications and treatments.

The orphan drug designation (ODD) is a result of positive findings from a phase 2 clinical trial which demonstrated a 100% clinical benefit rate in patients.

The treatment is indicated for patients with FRα-positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancers.

The data comes from 2 phase 4 trials, ENABLE and EMPOWER, which evaluated lanadelumab and the outcomes of treatment in adolescents with hereditary angioedema.

Findings show that magnetic resonance imaging and lumbar puncture are not always needed when managing complications of CAR T-cell therapies, but may be beneficial in certain cases.

The approval comes after positive results from the PhALLCON study, however, further research is needed to confirm immature event-free survival findings.

This approval makes atidarsagene autotemcel the only therapy to be approved in the US for early-symptomatic early juvenile metachromatic leukodystrophy.

The authors note that understanding the DNA methylation profile in leukemia can help predict whether or not a patient will respond to treatment.

The decision to vote in favor of idecabtagene vicleucel comes after positive phase 3 trial results demonstrating its efficacy compared with standard regimens.

Chimeric antigen receptor T-cell therapies were associated with higher incidences, grades of severity, and longer duration of cytokine release syndrome compared with bispecific antibodies.

In a phase 1/2 clinical trial, lisocabtagene maraleucel helped patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) achieve complete response rates.

This finding was evident in both examined groups and regardless of the patients' B-cell maturation antigen-directed therapy status.

The drug is indicated for patients 5 years of age and older with progressive familial intrahepatic cholestasis and cholestatic pruritus in pediatric patients with Alagille syndrome.

The combination of GZ17-6.02 and bortezomib reduced of HDAC, and as a result ATG13 phosphorylation was enhanced, BAK levels increased, and BCL-XL levels were reduced.

The findings also indicated that individuals who experienced significant adverse events in childhood were approximately 17% more likely to experience adverse events in adulthood.

The early phase clinical trial, which ended prematurely in 2013 due to funding challenges, further illustrates disparities in health equity in clinical research.

This initiative aims to raise worldwide awareness of the disease while trying to strengthen the connection between members of the multiple myeloma community.

The IVIG therapies were previously approved for 4-week room temperature storage conditions of 25º Celsius during the first 24 months of shelf life.