(Getty Images) The average American has 7.1 social media accounts, according to industry statistics. Yet while YouTube and TikTok are no substitutes for sound medical advice, doctors say there’s a ...

Children with sickle cell disease are at higher risk of developing sleep-disordered breathing, which may increase morbidity. Hydroxyurea therapy for children with sickle cell disease (SCD) has been ...

Early AATD diagnosis is crucial to prevent progressive lung damage and associated pathologies. Patients with alpha-1 antitrypsin deficiency (AATD) should be screened for vascular pathology, according ...

Life expectancy in DMD has improved over time. Previous estimates pointed to a life expectancy of 25 years, with more recent data reporting a life expectancy of 31.7 years (95% CI, 27.4-36.0). 3,4 A ...

Which of the statements below about the genetics of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) are correct? Select one of the options for each question, then submit your ...

The new formulation offers and additional choice and ease of use for patients with SMA. The US Food and Drug Administration (FDA) approved the New Drug Application for risdiplam tablets, sold under ...

Sodium-glucose co-transporter 2 inhibitors (SGLT2is) may be associated with a more favorable prognosis in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), according to a new ...

Researchers from Japan reported the case of a patient who was incidentally diagnosed with hereditary transthyretin amyloidosis (ATTR) during video-associated lung surgery to treat lung cancer. The ...

Which of the statements below about the pathophysiology of fetal and neonatal alloimmune thrombocytopenia (FNAIT) are correct? Select one of the options for each question, then submit your answers to ...

A systematic diagnostic approach should be used to differentiate AAV from multiple myeloma. An elderly patient who was initially suspected of having antineutrophil cytoplasmic antibody ...

The abnormal turnover and dynamics of mitochondria in skeletal muscles are associated with disease progression in spinal muscular atrophy (SMA) in a mouse model of the disease, according to a new ...

Developmental motor delay has frequently been reported as a risk factor for persistence of TLK, so postural control is crucial for attaining TLK resolution. Researchers in the Netherlands have ...