The project addresses a long-standing gap in the field: the lack of a clear human reference standard for muscle regeneration. A new research collaboration between muscular dystrophy organizations aims ...

Normalization of C1 inhibitor activity and C4 levels supported reclassification to type 3 disease after years of presumed type 2 angioedema. Reclassification of hereditary angioedema (HAE) subtype ...

The rarity of generalized pustular psoriasis is associated with limited clinical experience and thus creates difficulty in the assessment of therapeutic proposals. The Psoriasis Working Group has ...

Data showed berotralstat was well tolerated, maintained a consistent safety profile, and reduced monthly attack rates, with nasopharyngitis being the most commonly reported side effect. Berotralstat, ...

A banner welcomes delegates to the American College of Rheumatology’s 2025 conference in Chicago (Photo by Larry Luxner) The American College of Rheumatology awarded its 2025 Presidential Gold Medal ...

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 ...

Allogeneic stem cell transplantation and targeted therapy conferred the longest median survival after HMA failure. Stem cell transplantation and targeted therapies were associated with markedly ...

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 ...

The overall response rate of transfusion-dependent patients was 56% while that of transfusion-independent patients was 46%. Momelotinib treatment leads to clear clinical benefits in patients with ...

Results from a 2-sample Mendelian randomization analysis showed that there was a significant association between primary biliary cholangitis (PBC) and osteoporosis. Primary biliary cholangitis (PBC) ...

The increase in overall prevalence of mast cell disorder diagnoses was primarily driven by an 8-fold increase in use of the ICD-10 code D89.40, which lacks specific, validated diagnostic criteria. The ...

Kerrie Walters, who has MS, and her service dog, Wooldridge, at home in Grants Pass, Oregon (Credit: Jennifer Butler Photography) Kerrie Walters first heard of PoNS in a chance encounter with TV ...