Awareness of the key symptoms of NF1 can help in early diagnosis. A young male patient was diagnosed with neurofibromatosis type 1 (NF1) after undergoing detailed clinical investigations, according to ...

Nerandomilast is a first-in-class preferential inhibitor of phosphodiesterase 4B (PDE4B), shown to exert both immunomodulatory and antifibrotic effects. The orally administered therapy demonstrated a ...

In terms of glucocorticoid remission, the rituximab cohort achieved better results (52.1%) compared with the intravenous cyclophosphamide group (30.9%). Rituximab was found to be significantly ...

Ivabradine treatment slowed resting heart rate and reduced the levels of N-terminal prohormone of brain natriuretic peptide (NT-proBNP) in some patients with ATTR. MINNEAPOLIS—The sodium channel ...

Emerging therapies such as bezuclastinib and elenestinib show promise in improving disease control with potentially fewer toxicities. Other novel strategies, such as immune-based therapies, are under ...

Patients with autoimmune nodopathy more frequently had blood–cerebrospinal fluid barrier dysfunction and total intrathecal immunoglobulin G synthesis than patients with chronic inflammatory ...

The potential of vitamin D as an add-on therapy lies in its immunomodulatory effect on T cell-mediated and humoral immune responses, complement activation, cytokines, and inflammasomes. Vitamin D ...

Health regulators in the US are leading a push away from animal models in preclinical studies of potential disease treatments. While animal models are likely to still have a place in translational ...

More than half (52.4%) of the patients had target joints, with the mean number per patient ranging from 1.3 to 2.3, irrespective of hemophilia type or inhibitor status. In general, patients receiving ...

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

The patient recovered from surgery with no respiratory complications or any exacerbation in the symptoms of CIDP. Researchers from Iran presented the case of a patient with chronic inflammatory ...

After a decade of searching for an answer to what was causing progressive pain and weakness in my body, I was finally diagnosed with Pompe disease. Pompe, a rare genetic disorder, has long presented a ...