The care team individualized mechanical ventilation by adjusting tidal volume and closely monitoring driving pressure. Standard lung-protective ventilation (LPV) strategies can be difficult to ...

The authors observed that patients with extreme thrombocytosis had better overall survival (OS) than those with lower platelet counts. The presence of thrombocytosis in patients with myelofibrosis (MF ...

Smoking and frequent exacerbations increased lung function decline in both groups, but particularly in those with the PiZZ genotype. Patients with the PiZZ genetic form of alpha-1 antitrypsin ...

Mast cells can store IL-6, which can be released rapidly following mast cell activation or more slowly for a severe mast cell activation attack. A delayed severe mast cell activation (MCA) event has ...

Jerry Lewis, one of the most famous comedians of the 20th century, was a tireless advocate for people with muscular dystrophy. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews ...

In the US real-world data, the cumulative incidence of infection was 67.3%, that of serious infection was 8.4%, and the cumulative incidence of sepsis was 4.9%. Infections are a major comorbidity ...

A total of 11 patients with MF had a mutation in the DNMT3A gene. This finding suggests that somatic nondriver mutations such as this one could potentially be included in thrombosis risk scores in the ...

I recently returned home from the World Orphan Drug Congress 2025, and I am still excited and motivated by what I experienced. Not only did I attend, but I was also on a panel speaking with other ...

There are a few debated issues about what one is at risk for if the person has alpha-1 antitrypsin deficiency (AATD). Some things are known for sure, such as the risk of lung or liver disease and ...

It’s 2:25 am as I sit down to write this column. I’ve been awake for almost 2 hours and unable to go back to sleep. I had an immune thrombocytopenia (ITP) relapse, something I’ve been afraid of ...

Patients with Friedreich ataxia (FA) have high levels of cardiac troponin T in their serum suggesting an association with the severity of hypertrophic cardiomyopathy but there is no association ...

Study results revealed that almost half of all participants experienced poor sleep quality and insomnia during attack-free periods, and sleep scores worsened during attack periods. Individuals with ...