Neuromyelitis optica spectrum disorder (NMOSD) and multiple sclerosis (MS) have a negative impact on employment, found a study published in the journal Multiple Sclerosis and Related Disorders. “The ...

IgG4-related orbital disease should be considered as a possible diagnosis in cases of orbital inflammatory disease. A study characterizing orbital inflammatory disease (OID), a differential diagnosis ...

The FDA approval of casimersen was based on the increased dystrophin production observed in patients with DMD who were treated with the drug. 1 The increased production of a functional protein may be ...

The most frequently mutated genes were associated with epigenetic regulatory mechanisms, namely TET2, ASXL1, and DNMT3A. Next-generation sequencing (NGS) identified 14 gene mutations with diagnostic ...

Nearly two-thirds of adult patients on crinecerfont achieved glucocorticoid doses within the physiologic range, compared to just 18% on placebo. The US Food and Drug Administration (FDA) has approved ...

As of September 2024, a patient with CLL has sustained a complete response after beginning methionine restriction in 2022. A patient with chronic lymphocytic leukemia (CLL) achieved complete response ...

The 2D segmentation models showed best performance for the iliac bone marrow. Researchers from the United States developed 4 different deep-learning U-Net models that could effectively automate bone ...

Chronic lymphocytic leukemia (CLL) is a mature B-cell hematological malignancy characterized by progressive clonal proliferation of B lymphocytes in the peripheral blood, bone marrow, and lymph nodes.

The serious side effects of phototherapy, such as overheating and dehydration, are driving the need for adjuvant therapies. A new literature review has observed promising results from the use of zinc ...

In CIDP, SOC therapies (immunoglobulins/corticosteroids) are burdensome, have variable efficacy, and significant side-effects. Two phase 3 clinical trials, MOBILIZE ...

The expression of AQP4 In the respiratory epithelium suggests that lung involvement might be an unrecognized feature of NMOSD. Patients with neuromyelitis optica spectrum disorder (NMOSD) should be ...

Luke Chen, MD, MMEd, is a clinical hematologist and professor of medicine at Dalhousie University in Nova Scotia, Canada. Rare Disease Advisor correspondent Tori Rodriguez interviews clinical ...