Jaime Rosenberg

As of September 2022, all but 2 states had implemented newborn screening programs for spinal muscular atrophy (SMA), accounting for 98% of births in the United States.

With most previous studies on ocular myasthenia gravis stemming from investigations the neurology field, the present investigators came to their conclusion via neuro-ophthalmic analysis.

The researchers note that this finding adds valuable insight for individualized treatment decisions in scenarios where the role of thymectomy is not fully clear.

The systemic review and meta-analysis, believe the researchers, is the first of its kind to characterize the prevalence of depression among these patients.

Patients who received nirmatrelvir plus ritonavir (Paxlovid) had lower risk of both hospitalization within 14 days and death within 28 days compared with patients who did not receive the treatment.

The importance of identifying rare pathogenic mutations and genetic risk score among patients was explored in this new analysis, with goals that included reducing mortality and early disease detection.

A new analysis has determined the cost-effectiveness of nirmatrelvir/ritonavir (Paxlovid), granted emergency use authorization by the FDA, across various scenarios of vaccination status and risk for severe disease.

Data from the secondary analysis show that the improvements seen in health-related quality of life (HRQOL) translated into clinical improvements.

The researchers hypothesize that SARS-CoV-2 infection may have triggered thymic inflammation, spearheading initial expression of muscle-like epitopes and T-cell dysregulation.

The findings, which the researchers say highlight the importance of early diagnosis and initiation of treatment, come amid efforts to determine efficacy trends among the several treatment options for the condition.

The findings, say the researchers, confirm that repeated intrathecal injections of the gene-targeting therapy are safe, adding to the limited data on the long-term effect and safety of the treatment approach in neurological diseases.

Though a large proportion of patients receiving oral immunotherapy (OIT) for food allergy report gastrointestinal (GI) symptoms, a knowledge gap remains for the best approach to determine the underlying etiology and manage symptoms.

Drawing on findings from other countries, including the United States, the group is highlighting the need for a newborn screening program to mitigate these current barriers.

Based on their retrospective findings, the researchers suggest the diet is a potentially effective option for these patients who may face challenges with other elimination diets.

This paper follows the 10-year remission mark for the first child treated with CD19-targeted CAR (chimeric antigen receptor) T-cell therapy for B-cell acute lymphoblastic leukemia (B-ALL), adding promise to the notion that the technology can provide long-term durable remissions in patients who are highly refractory to chemotherapy.

A review of available literature, although limited, has pointed to a link between pediatric psoriasis and anxiety and depression. Previous research has made a clear association between psoriasis and mood disorders in adults.

Data on the use of biologics for psoriasis among patients with a history of cancer have remained sparse. However, new findings provide the largest amount of real-world data on the use of the biologic secukinumab in these patients.

With 60% to 75% of patients with rheumatoid arthritis (RA) developing other long-term conditions throughout their lifetime, the researchers underscored the importance of using multimorbidity in risk stratification of patients with RA based on their findings.

Generally, the studies suggested a decreased risk of rheumatoid arthritis (RA) associated with fish and vegetable consumption, as well as Mediterranean-style diets, although the researchers noted that these studies used varying methods and had varying results.

Their findings, say the researchers, offer useful insights into the outcomes and risks of severe disease and mortality in patients who have multiple myeloma (MM) or amyloidosis (AL) with underlying monoclonal gammopathy who contract COVID-19.

Researchers underscore a need for prospective data, noting that women with chronic kidney disease (CKD) tended to have higher rates of preterm delivery and lower birthweight.

Stemming from recent data on a potential link between major depressive disorder (MDD) and inflammation, research is starting to turn toward alternatives that can treat the disease through anti-inflammatory effects, including ketamine, cannabinoids, and psychedelics.

Detailing findings from the secondary end points of the FIREFISH trial at 24 months, the researchers found that risdiplam treatment was associated with continued improvements in motor function and the achievement of development motor milestones.

A new study has found that a drop in serum inorganic phosphate (iP) was an early predictor of severe cytokine release syndrome (CRS), suggesting that monitoring serum iP levels could help promote safer management of the most common toxicity associated with chimeric antigen receptor (CAR) T-cell therapy.

Using data from patients with spinal muscular atrophy (SMA) type 1 and 2, researchers observed that chitotriosidase 1 levels in cerebral spinal fluid (CSF) changed over time after treatment with nusinersen, suggesting that the change could be indicative of treatment response.

The observational study found that 25% of deaths were premature and that mortality associated with rheumatoid arthritis (RA) resulted from cardiovascular, cancer, and respiratory causes

Zanubrutinib is the latest of 3 Bruton tyrosine kinase (BTK) inhibitors approved for rare B-cell malignancies, such as Waldenström macroglobulinemia, a non-Hodgkin B-cell lymphoma (NHBCL); as a next-generation BTK inhibitor, zanubrutinib is designed to have less off-target effects.

While chimeric antigen receptor (CAR) T-cell therapy has offered a new option and improved outcomes for patients with advanced hematologic malignancies, the treatment approach has been associated with toxicities such as cytokine release syndrome (CRS), neurotoxicity, and hematotoxicity.

Recent research suggested the idea of combining chimeric antigen receptor (CAR) T-cell therapy and Bruton’s tyrosine kinase (BTK) inhibitors for certain patients with advanced mantle cell lymphoma (MCL).

The retrospective findings showed that searching for Staphylococcus aureus and its enterotoxins (SE) when initially screening patients for asthma can help with identifying a targeted treatment option by improving phenotyping and predicting comorbidities.