Gillian McGovern, Associate Editor

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.

The study results also indicated that graft versus host disease status was associated with non-compliance following bone marrow transplantation.

In addition, the length of hospital admissions were greater in patients with hidradenitis suppurativa-associated cutaneous squamous cell carcinoma compared to other admissions.

Despite the belantamab mafodotin regimen improving overall survival in patients with relapsed or refractory multiple myeloma, the trial is ongoing to confirm the presented results.

The vice president and chief pharmacy officer at City of Hope explains how she serves as a mentor for women within health care, the importance of networking, and staying educated.

The vice president and chief pharmacy officer of City of Hope discusses obstacles women face in health care and how diversity, equity, and inclusion can be promoted within the space.

Patients with urothelial carcinoma who were treated with the nivolumab-based regimen had better OS and PFS compared with those who received cisplatin with gemcitabine alone.

The designation is for all patients with low-grade serous ovarian cancer, regardless of their KRAS status and after 1 or more prior lines of therapy.

The device conducts blood biomarker tests that accurately diagnose amyloid pathology and can assist in diagnosing Alzheimer disease in patients with cognitive impairment.

This designation makes CBL-514 the first drug to receive both Orphan Drug Designation and Fast Track Designation for the treatment of Dercum disease.

The next-generation SRI was previously granted an Orphan Drug Designation in November 2023 and its safety and efficacy are currently being evaluated in clinical trials.

According to the researchers, making changes to the daylight saving time system to accommodate heart health is unnecessary.

The regimen, which was used as second-line treatment in patients with drug-resistant TB, had a similar microbiological response to those with drug-sensitive TB taking first-line regimens.