Jared Kaltwasser

Investigators said patients experienced adverse effects in line with those expected for myeloablative conditioning with busulfan following treatment with betibeglogene autotemcel (beti-cel; Zynteglo; bluebird bio).

The single-center report showed the therapy had a favorable safety profile and manageable side effects in children with relapsed or refractory (R/R) acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS).

Patients continued taking their medications for pulmonary arterial hypertension (PAH) when symptoms appeared to improve, according to a new survey.

A systematic review has found a potential link between edentulism and sleep apnea risk, although the authors said differences in study designs prohibited a meta-analysis.

The new report is an attempt to provide comprehensive safety data on the rapidly changing treatment landscape for relapsed or refractory chronic lymphocytic leukemia (CLL).

New research suggests previous assumptions about the pathogenesis of spinal muscular atrophy (SMA) are inadequate.

Investigators say they may have found a new therapeutic target for patients with double-expressor diffuse large B-cell lymphoma (DLBCL).

Investigators used 3-dimensional quantitative computed tomography to more accurately assess patients’ bone mineral density.

New data suggest the frequency of damage and repair processes correlates with disease severity and disability in multiple sclerosis (MS).

The therapy reduced inflammation and appeared to slow disease progression in models, suggesting it could have similar impacts in humans.

The gene therapy improved annualized bleed rates, though a handful of study participants resumed factor IX prophylaxis.

Physicians also differed from caregivers when asked how many hours of caregiving patients required each week.

New research helps explain why a subset of patients with relapsing/remitting multiple sclerosis (MS) experience long-term heart rate slowing after taking fingolimod.

Various therapies have different impacts on brain volume loss in patients with relapsing multiple sclerosis (MS), a new study has found.

A new subgroup analysis of patients with muscle-specific tyrosine kinase (MuSK) autoantibody-positive (Ab+) myasthenia gravis (MG) shows rozanolixizumab (Rystiggo) contributes to meaningful symptom improvement.

Investigators said the unique features of chimeric antigen receptor (CAR) T-cell therapy may warrant novel approaches to cost-effectiveness analysis.

Investigators say thrombospondin-4 levels were reduced in patients with symptoms of spinal muscular atrophy (SMA), but levels of the protein increased after therapy.

Investigators say adding RNA interference (RNAi) therapy to current treatment modalities may improve outcomes in patients with acute myeloid leukemia (AML) with internal tandem duplication in the fms-like tyrosine kinase 3 (FLT3) gene.

The study is among the first to look at the potential impacts of clonal dynamics on patients with lower-risk myelodysplastic syndromes (MDS).

A comparison of the second-generation Bruton tyrosine kinase (BTK) inhibitor acalabrutinib to the first-generation ibrutinib shows the former had lower rates of adverse events in a real-world setting.

Insulin resistance plays a role in many diseases. The new research provides insights into why insulin resistance appears to be associated with neurodegenerative disorders.

Most patients diagnosed with chronic-phase chronic myeloid leukemia (CP-CML) who achieved a deep molecular response on tyrosine kinase inhibitor (TKI) therapy were able to avoid relapse despite discontinuing therapy.

However, an analysis suggested that androgenetic alopecia is not caused by hypothyroidism.

A study suggests most patients with myasthenia gravis (MG) can take statins without experiencing symptom worsening.

Health plans would see savings faster if they switched to etranacogene dezaparvovec sooner, the investigators found.

New research shows how repetitive nerve stimulation and single fiber electromyography can help diagnose disorders such as myasthenia gravis.

Though magnesium has long been seen as a potential sleep aid, the authors say the new form of magnesium salt may provide a clearer benefit.

Researchers say machine learning advances make it possible to use more sophisticated body composition analyses to assess risk of mortality from idiopathic pulmonary fibrosis (IPF).

A new report based on patient focus groups suggests many clinicians can do more to help patients with sickle cell disease (SCD) feel heard and understood.

The tentative approval cannot take effect because a competing product was granted market exclusivity.