Key outcomes included high ILD prevalence, postmyopathic PPF in 13.5% of the cohort, higher rates of rapidly progressive ILD and transplantation in postmyopathic PPF, greater rituximab use in PPF, and ...

At 4 weeks post-surgery, the patient's anterior tibialis and quadriceps strength had improved from 2 to 3 out of 5 preoperatively to 4 out of 5. Routine fluoroscopy and high-magnification ...

Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...

Efgartigimod demonstrated clinically meaningful benefit and acceptable tolerability in patients with double-seronegative generalized MG. Rare Disease Advisor, a trusted source of medical news and ...

A qualitative matrix analysis allowed for the development of implementation strategies to improve the efficiency and effectiveness of the system. Researchers from the US identified an ideal system to ...

Baseline hemoglobin at diagnosis and patient age independently predict transfusion density, whereas other hematologic factors do not. A higher cumulative red blood cell (RBC) transfusion burden is ...

HDFN can cause adverse outcomes both prenatally and postnatally. Hemolytic disease of the fetus and newborn (HDFN) can be associated with both severe pre- and postnatal outcomes, according to a study ...

Notably, the CRL did not identify safety or efficacy concerns. Aquestive Therapeutics, Inc. announced that the US Food and Drug Administration (FDA) has issued a complete response letter (CRL) for ...

Nintedanib significantly reduced the decline in the forced vital capacity of patients with progressive pulmonary fibrosis (PPF). Treatment with nintedanib significantly slows the progression of ...

Chimeric antigen receptor (CAR) T cells based on the proximal region of mesothelin completely eradicated CCA tumors in 3 different mouse models. Mesothelin, a tumor differentiation antigen that is ...

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 ...

Between 2001 to 2011, annual age-adjusted incidence increased from 0.55/100,000 to 0.78/100,000, demonstrating an increasing trend with age. Incidence peaks in individuals over 70 years of age ...