There is significant variability in terms of management practices across Canadian medical centers in terms of red blood cell (RBC) and platelet transfusions in preterm newborns, according to a study ...

In a letter to the editor of the European Respiratory Journal, Newman and colleagues presented the findings of a study demonstrating that an electronic patient-reported outcome measure (ePROM) for ...

Next-generation sequencing (NGS) of fetal cell-free DNA from maternal plasma provides a highly accurate and noninvasive approach to determine fetal erythrocyte and platelet antigen status in ...

Patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) who were treated with tafamidis for a year showed stabilization of several cardiac biomarkers, according to a study published in ...

Risdiplam treatment for 2 years leads to improvements in quality of life in patients with spinal muscular atrophy (SMA), according to a new study published in the scientific journal Neuromuscular ...

Researchers from Brazil developed a new clinical score to estimate the probability of neuromyelitis optica spectrum disorder (NMOSD) in patients presenting with optic neuritis.. The easily accessible ...

Weight loss may be intensified by treatment-related side effects of diffuse large B-cell lymphoma (DLBCL), such as loss of appetite, diarrhea, 1 vomiting, 1,2 nausea, and changes in taste and smell. 2 ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Antonella Favit-Van Pelt, MD, PhD, chief medical officer at Helius Medical, on the potential of neuromodulation to treat people ...

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a disorder characterized by progressive, symmetrical muscle weakness and disturbances in the distal upper and lower extremities. While it is ...

Patients with the PiZZ genetic form of alpha-1 antitrypsin deficiency (AATD) have more aggressive disease and loss of function than individuals with the PiSZ form, according to results published in ...

Hereditary transthyretin amyloid polyneuropathy (ATTR-PN) due to a Ala97Ser(p.Ala117Ser) mutation in the TTR gene is relatively rare in South Mainland China, and many cases were misdiagnosed as ...

The advent of chimeric antigen receptor T-cell (CAR-T) therapy for relapsed or refractory diffuse large B-cell lymphoma (DLBCL) has led to improved survival across patient subgroups, according to ...