Unlike the pivotal studies of ruxolitinib or fedratinib, phase 3 trials for pacritinib (PAC) enrolled patients with INT-1 risk. During the studies, a spleen volume reduction of at least 35% (SVR35) ...

The core care team for TK2d may include a neuromuscular neurologist, pulmonologist, nutritionist, physical therapist, and orthopedist. A support care team may include specialists such as a ...

Recently, I wrote about my regular sense of the need for another infusion for alpha-1 antitrypsin deficiency (AATD). After about 5 days following an infusion day, I tend to start feeling more out of ...

Testosterone levels positively correlated with hepatic fibrosis in patients with Wilson disease and PCOS. Polycystic ovary syndrome (PCOS) can be a frequent comorbidity in women with Wilson disease ...

Thymidine kinase 2 deficiency (TK2d) affects many organs and systems, leading to a variety of clinical features. However, the main clinical features of TK2d are progressive muscle weakness, dysphagia, ...

There was an increased risk of atrial fibrillation in the patients regardless of the presence of heart failure or other common atrial fibrillation causes. Individuals with alpha-1 antitrypsin ...

Based on their findings, the researchers developed a new scoring system, IPSS-R-I, which demonstrated superior performance compared to the original IPSS-R system. Immunohistochemical profiling of bone ...

In total, 139 independent pharmacist or registered nurse visits took place, allowing clinicians to focus on other patient needs. Pharmacist and registered nurse (RN)-led management of venetoclax ...

The annualized relapse rate was 0.069 after the start of treatment with satralizumab and 0.445 within 52 weeks before the start of treatment with the drug. Treatment with satralizumab is associated ...

Neutropenia was a dose-limiting toxicity in patients who were Duffy positive and Duffy null, but the effect was more prominent in the Duffy-null group. Individuals with the Duffy null phenotype ...

Patients with pulmonary arterial hypertension (PAH) can develop several complications with the progression of the disease or as a result of hemodynamic imbalance. Some of these complications include ...

Patients with anti-AQP4 antibody-positive NMOSD who were current smokers and had vascular risk factors had a higher risk of residual disability from onset attacks. Smoking has a detrimental effect on ...