AJMC Contributor

New data from the single-center, open-label extension EDITA-ON study suggest that early treatment for pulmonary arterial hypertension (PAH) could benefit patients with systemic sclerosis and early pulmonary vascular disease.

A recent study showed that the Myasthenia Gravis (MG) Symptoms PRO scales viably measure core symptoms of the disease.

Beremagene geperpavec-svdt (B-VEC) entered the market in 2023 as the first approved corrective treatment for dystrophic epidermolysis bullosa (DEB), a rare genetic disease affecting the skin and nails that is caused by mutations in the COL7A1 gene.

The biomarker, G-protein-coupled receptor 176 (GPR176), may be a driver in the progression of ovarian cancer and potential target for gene therapy, suggested researchers of a new study.

Researchers collected feedback from 20 clinicians implementing a telehealth serious illness conversation (SIC) with their patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

The researchers noted that most genomic analyses have traditionally focused on commonly mutated genes, which can pinpoint mutations occurring most frequently but does not account for the extent to which these mutations impact cancer cell survival and proliferation.

In a recently published systematic review, researchers determined that VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome typically presents in men, with atypical morphology and with systemic symptoms.

In addition to ensuring residency programs provide educational and practical opportunities for addressing skin of color (SOC) hair-related concerns, prioritizing having residents of diverse backgrounds can help close knowledge and comfort gaps, suggest researchers.

Of 7 subtypes identified 10,000 melanoma cells, C4 Melanoma COCO1A was the only to show indications of being more sensitive to natural killer (NK) and T cells.

With an emerging hypothesis that inflammation of the skin prompts intestinal remodeling through various mechanisms, researchers explored the role of inflammatory signals, microbiome alterations, metabolites, and the nervous system.

Early findings from a new preclinical study indicate that dual blockade of aurora-A kinase and PD-L1 signaling can inhibit tumor growth in triple-negative breast cancer (TNBC), treatment for which has historically remained limited.

Notably, adolescents and young adults (AYA) living with type 1 diabetes (T1D) who reported more comfort with self-management tended to have worse glycemic control.

Data from this new study suggest that tailoring induction therapy for acute myeloid leukemia (AML) based on cytarabine pharmacogenomic 10–single-nucleotide variant score could better treatment for these young patients, particularly those who are Black.

Patients receiving chlorthalidone had lower rates of major cardiovascular (CV) events and noncancer-related deaths vs those receiving hydrochlorothiazide.

Although further research is needed, these new findings suggest that using the 2 intranasal sprays is an effective option for patients with myasthenia gravis (MG) who experience eyelid drooping.

The data come from the observational Cost of Hemophilia in Europe: A Socioeconomic Survey II (CHESS II) study of 288 Spanish patients with hemophilia A and B, which showed certain differences between disease subtypes but overall similar trends in disease impact.

An abstract presented at the 2024 American Society of Clinical Oncology annual meeting showed that patients treated with zanubrutinib or acalabrutinib had more favorable safety and efficacy outcomes.

Researchers say their findings will help inform treatment protocols for this patient population, as questions remain about optimal duration of these treatments following first-line treatment with pyridostigmine.

The proof-of-concept study showed that antisense oligonucleotides developed by the researchers successfully restored cellular development and brain cell function in patients with the disease characterized by multiorgan dysfunction.

Myasthenic crisis has been estimated to occur in up to 30% of patients with myasthenia gravis after thymectomy.

A retrospective study of over 100 pediatric and adolescent patients showed that providing virtual training for insulin pump use was both safe and effective.

Prospective data from patients attending a rural practice in West Virginia between 2016 and 2023 show that a quality improvement program that followed guideline recommendations and was tailored to specific patient needs reduced preventable bleeds and lowered costs.

Survey data from over 200 patients showed that treatment discontinuation was lower for those taking mycophenolate or methotrexate than for those taking azathioprine for their myasthenia gravis (MG).

In addition to her advanced age and obesity status, the patient had a history of pulmonary embolism managed with an inferior vena cava filter, increasing her risk of thrombosis.

Among over 60,000 nursing home residents who initiated antihypertensive medication, rates of excess fractures due to falls per 100 person-years were as high as 5 among certain patient groups, such as those with dementia and high blood pressure (BP).

Data from the study showed that exa-cel prevented vaso-occlusive crises in all but 1 of the 30 evaluable patients for at least 12 months.

Over a median follow-up time of nearly 3 years, three-fourths (72.8%) of patients with myasthenia gravis (MG) were hospitalized and half visited the accident and emergency department.

Self-reported data from adult and pediatric males with hemophilia A show that burden of the disease persists regardless of severity, highlighting a need for improved prophylactic treatment.

The randomized trial found that reducing sitting time over a 6-month period was association with reduced systolic blood pressure (BP) among a group of older patients with obesity and high rates of hypertension.

In their preclinical model, the researchers found efficacy both in vitro and in vivo by using CRISPR-Cas9 to mimic porphyria and combining the technology with light therapy.