Lower rates of chronic and acute GVHD, infections, and nonrelapse mortality indicate a substantially reduced toxicity burden. In patients with myelodysplastic syndrome (MDS) and acute leukemias ...

Amy Comstock Rick of the US Food and Drug Administration speaks at the 2026 Muscular Dystrophy Association Clinical and Scientific Conference (Photo by Riya Ajmera) Due to differences in the nature of ...

A mediation analysis revealed that 16.4% of nintedanib's effect on the change in forced vital capacity at week 52 was attributed to the treatment-induced reduction in the level of cancer antigen 125 ...

Treatment interruption led to biochemical relapse in 2 patients, but remission was restored after re-initiation of sutimlimab therapy. Real-world data from a small compassionate-use cohort at a single ...

Prompt discontinuation of the suspected drug is critical for recovery. Drug-induced immune hemolytic anemia (DIIHA) should be investigated in suspected cases of warm autoimmune hemolytic anemia (warm ...

The Reagan-Udall foundation was named for President Ronald Reagan and Congressman Morris “Mo” Udall from Arizona, each of whom had an incurable neurodegenerative disease. The Reagan-Udall Foundation ...

When I went out a few days ago, I noticed everyone had a runny nose or a cough. The weather here in Ohio has been chilly, about 30 °F (-1 °C), but we haven’t had our usual January extreme cold yet.

The authors argue that the long-held label of "uniformly lethal" may no longer be appropriate and can inadvertently bias clinical decision-making. Homozygous achondroplasia is traditionally ...

Baseline hemoglobin at diagnosis and patient age independently predict transfusion density, whereas other hematologic factors do not. A higher cumulative red blood cell (RBC) transfusion burden is ...

The low positive predictive value indicates that a positive diagnosis using planar imaging would result in an incorrect diagnosis of ATTR-CM 42% of the time. Calculating visual scores using ...

Among the 175 patients without atrial fibrillation at baseline, 41% developed new-onset atrial fibrillation over a median follow-up of 2 years. Over 50% of patients with transthyretin-mediated amyloid ...

The company has initiated a rolling BLA submission and expects to complete the process in the second quarter of 2026. Isaralgagene civaparvovec (ST-920), Sangamo Therapeutics’ investigational gene ...