Karen Jacobson-Sive

Patients with myasthenia gravis (MG) largely expressed negative emotions around MG treatment and symptom burden in patient focus groups and on MG social media discussion boards.

Most patients with pulmonary arterial hypertension (PAH) suffered from poor sleep quality, and dyspnea was a significant predictor of sleep quality, independent of disease severity, according to this study.

Rigorous surveillance of children at high risk for developing myelodysplastic syndrome (MDS) is especially warranted, according to researchers.

Perspectives of patients with spinal muscular atrophy (SMA) and their caregivers should be weighed before making decisions around SMA-enhancing treatments.

Rituximab-induced interstitial lung disease in patients with non-Hodgkin lymphoma presents with distinct clinical characteristics and bronchoalveolar lavage results.

The long-term response rate for zanubrutinib was better than ibrutinib in patients with relapsed/refractory chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL).

Oral function should be measured routinely to determine individual bulbar involvement stages in spinal muscular atrophy (SMA).

Patients with low-count chronic lymphocytic leukemia phenotype monoclonal B-cell lymphocytosis had a 1.86-fold greater risk of melanoma.

CNTN1 and NRXN3 clearly differentiated severe and milder forms of spinal muscular atrophy (SMA).

Three patients with RPE65-related retinopathy showed improved function in both eyes following gene augmentation therapy treatment with voretigene neparvovec.

Capivasertib, in combination with venetoclax, produced promising responses in preclinical models of diffuse large B-cell lymphoma (DLBCL).

B-cell activating factor receptor chimeric antigen receptor (CAR) T-cell therapy caused cytotoxic effects on all chronic lymphocytic leukemia (CLL) cell lines.

Patients with sickle cell anemia (SCA) and persistent albuminuria (PA) exhibited significantly higher levels of urinary angiotensinogen (AGT).

Immunosuppressants used in adeno-associated virus (AAV) gene therapy for monogenic disorders present an adverse effect profile that is consistent with high-dose steroid use in other conditions, according to a meta-study that analyzed immunosuppressive protocols.

Celiac disease presents a higher risk for developing malignant neoplasms, including small intestine, lymphoma, and skin cancers, but presents a lower risk for respiratory malignancies.

Donor lymphocyte infusion, both alone and in combination with azacitidine, restored CD34 levels in two-thirds of patients with a baseline CD34 donor chimerism of less than 80%.

Researchers have discovered 2 leukemia stem cell subtypes with distinct epigenetic and transcriptional signatures.

Recombinant Hirudin (rH) inhibited the polarization of M2-type macrophages and protease-activated receptor-1 (PAR-1) in diffuse large B-cell lymphoma (DLBCL).

The decremental response follows repetitive nerve stimulation (RNS) completed earlier in proximal muscles compared with distal muscles in a study of patients with myasthenia gravis (MG) and amyotrophic lateral sclerosis (ALS).

A series of portable respiratory function tests for patients with mild to moderate myasthenia gravis (MG) were found to be useful methods to diagnose previously unidentified at-risk cases for respiratory deterioration.

Patients with hypertension-related chronic kidney disease who increased daily fruit and vegetable consumption showed improved cardiovascular health and reduced blood pressure compared with participants who added baking soda to their diet or who received pharmacological-only treatment.

An exon-skipping technique using dual single-guide RNA/Cas9 ribonucleoproteins targeted at 3 novel COL7A1 exons with pathogenic heterozygous mutations achieved exon deletion rates of up to 95%.

Post–hematopoietic cell transplantation (HCT) prophylaxis using cyclophosphamide to stave off graft-vs-host disease had positive results compared with calcineurin inhibitor–based prophylaxis, regardless of whether the patient had a matched or unmatched donor.

The hospitalization rate for patients with pulmonary hypertension (PH) and interstitial lung disease (ILD) doubled over a 2-year period, and total health care costs more than doubled over the same period.

Liposomal daunorubicin and cytarabine (CPX-351) was effective in relapsed acute myeloid leukemia, but long-term follow-up is needed to determine the extent of the agent's cardiotoxicity.

Pirtobrutinib led to improved outcomes compared with venetoclax in patients with relapsed chronic lymphocytic leukemia (CLL) following covalent Bruton tyrosine kinase inhibitor therapy.

A post-hoc analysis showed that zanubrutinib resulted in fewer adverse events compared with ibrutinib.

This meta-analysis study will estimate the global and regional burden of HIV-associated non-Hodgkin lymphoma to aid the development of prevention and control strategies.

Patients with myasthenia gravis who received treatment within a year from diagnosis were generally in accordance with guideline suggestions.

More than 60% of complement inhibitor–naive patients with acetylcholine receptor–positive generalized myasthenia gravis (MG) in diverse clinical populations had a clinically meaningful reduction in MG activities of daily living scores.