Despite the suboptimal imaging conditions, TTE and CMR measurements of LVEF demonstrated high agreement, with a mean difference of 1.6%. Transthoracic echocardiography (TTE) and cardiac magnetic ...

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 ...

An IDEAL-IQ FF threshold of 52.3% in the vastus lateralis accurately identified loss of ambulation with 92.5% sensitivity and 100% specificity. Findings from a recent study published in the Journal of ...

Ambulatory boys with DMD treated with delandistrogene moxeparvovec maintained NSAA scores above baseline at year 3. Sarepta Therapeutics announced positive 3-year functional outcomes from the EMBARK ...

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 ...

Castleman disease’s higher mortality and invasiveness require prompt recognition, while IgG4-ROD’s benign yet recurrent nature supports immunomodulatory therapy and long-term follow-up. Chinese ...

The liver stiffness measurement was reduced by a median of 0.2 kPa, with a percent reduction of 2.9% in patients with PBC treated with seladelpar over 36 months. Most patients with primary biliary ...

A multidisciplinary approach is necessary for the deep phenotyping and proper clinical management of individuals with primary mitochondrial diseases. A large percentage of patients (60%) with primary ...

The treatment could potentially help patients reduce the use of glucocorticoids and immunosuppressants, the authors of the study said. The safety and efficacy of efgartigimod for the treatment of ...

Biochemical response rates were sustained through week 182 in patients with PBC treated with elafibranor, and the proportion of patients with normal alkaline phosphatase levels remained consistent.

The consequences of cystic fibrosis (CF) are extensive and affect almost every organ system. The clinical picture is broad and may present with acute or chronic manifestations. Gastrointestinal and ...

Professor Aya Abrahamov and two Palestinian women with Gaucher disease share a light moment at Shaare Zedek Medical Center in Jerusalem, Israel (Photo by Ari Zimran) Shaare Zedek’s Gaucher unit is the ...