Which of the statements below about the clinical features of Friedreich ataxia (FA) are correct? Select one of the options for each question, then submit your answers to see how well you scored and ...

Necrotizing and crescentic glomerulonephritis arises in all 3 AAV subtypes: GPA, MPA, and EGPA. According to findings from a study recently published in the journal Kidney International, among ...

The study will measure the proportion of pregnancies without intrauterine transfusion, hydrops fetalis, or fetal loss/neonatal death. Phase 3 of the AZALEA study, the first placebo-controlled, ...

The study will assess changes in disease activity and adverse events, including hospitalizations and their duration. A new phase 1 study is set to evaluate the safety and activity of rituximab in ...

HPA genotyping is an important investigation in assessing the underlying cause of a neonate presenting with severe thrombocytopenia. A 34-year-old female (gravida 6, para 4) presented at 31 weeks of ...

Internationally, next to matching for D, matching for K and other Rh antigens (C, c, E, and/or e) is often applied to prevent HDFN, and "cEK matching" has been strongly advocated in The Netherlands ...

Benralizumab helped nearly 60% of patients with eosinophilic granulomatosis with polyangiitis achieve remission. Results from the phase 3 MANDARA trial demonstrated efficacy for benralizumab (Fasenra ...

As a patient with alpha-1 antitrypsin deficiency (AATD), I didn’t always have an aversion to strong scents. In fact, I enjoyed having perfumes and scented candles as a teenager. However, now that I’m ...

The US Capitol building in Washington, DC (Photo by Larry Luxner) The Rare Pediatric Disease Priority Review Voucher program allows FDA review of an application to be completed in 6 months instead of ...

The study demonstrated that individuals with CANVAS had significantly smaller nerve cross-sectional area (CSA) across various nerves. Patients with Friedreich ataxia (FA), neuropathy, and vestibular ...

FNAIT may occur when paternally inherited human platelet antigens (HPAs) on the surface of fetal platelets are incompatible with those of the mother and recognized as foreign by the mother’s immune ...

Findings of recent studies confirm an effect of the disease on skeletal muscles and support the idea that of progressive mitochondrial damage drives the progression of the disease. The effects of ...