The adjusted mean change in forced vital capacity at week 52 was –107.1 mL in patients given placebo, –61.2 mL in patients treated with 9 mg of nerandomilast twice a day, and –64.9 mL in patients ...

The FDA granted Fast Track Designation to coramitug for ATTR-CM as it advances in the ongoing phase 3 CLEOPATTRA trial. The US Food and Drug Administration (FDA) has granted Fast Track Designation to ...

In the ADAPT SERON trial, treatment with efgartigimod led to rapid, significant, and sustained improvements in symptoms such as speech, vision, swallowing, and physical function. The US Food and Drug ...

There were 7 cases of PAH temporally associated with bevacizumab in the French Pulmonary Hypertension Registry, but most had predisposing conditions. Bevacizumab exposure does not seem to lead to the ...

Genetic analysis of 2 independent cohorts showed celiac disease increases the odds of pulmonary arterial hypertension, with a meta-analysis odds ratio of 1.136. Celiac disease increases the risk of ...

At a median follow-up of 22.4 months, event-free survival was 71.4% and overall survival was 78.6%. Hematopoietic stem cell transplantation (HSCT) is both possible and effective in adult patients with ...

The model had an area under the receiver operating characteristic curve of 0.83 in a high-risk subgroup of patients with conditions that mimic ATTR-CM. An artificial intelligence model trained to ...

Treatment was well-tolerated, with a safety profile consistent with prior treprostinil studies and no new safety signals. Nebulized treprostinil (Tyvaso ®) demonstrated superiority over placebo in ...

Clinical trials showed significant reductions in lesion burden, symptoms, and mast cell density. Avapritinib produced consistent and clinically meaningful improvements in cutaneous manifestations ...

The high-output heart failure cohort was characterized by an increased frequency of secondary MF and lower baseline hemoglobin levels. Echocardiographic high-output heart failure in patients with ...

Despite a phase 3 trial that narrowly missed its primary endpoint, Sarepta is pursuing traditional approval for the 2 DMD therapies. Sarepta Therapeutics will submit supplemental new drug applications ...

Preclinical findings show DYNE-401 enables lower and less frequent dosing while targeting both skeletal muscle and the CNS in Pompe disease. Even when administered every other month, DYNE-401 ...