The FDA has set an action date of August 21, 2025, under the Prescription Drug User Fee Act. The agency previously granted Orphan Drug Designation for donidalorsen in 2023. The US Food and Drug ...

Three-dimensional computed tomography complements fetal ultrasonography in detecting early signs of fetal skeletal dysplasia during pregnancy. Three-dimensional computed tomography (3D-CT) is a highly ...

Early detection, differentiation, and intervention via regular screening are essential to managing the optical anomalies that can occur in this condition. The first known case of a patient with ...

Despite differences in severity, all patients with recurrent ITP eventually achieved platelet remission, with similar time-to-remission trajectories for the first and recurrent episodes. A recent ...

A significant number of patients responded that genital attacks negatively affected their sexual lives. The negative feelings about sexual life might be attributed to HAE itself, not to the site of ...

Acoramidis significantly reduced death and cardiovascular-related hospitalization as early as 3 months after the start of therapy. The US Food and Drug Administration (FDA) approved a new oral ...

Treatment with eplontersen led to improvements in autonomic function, quality of life, and mody mass index. Eplontersen led to improvements in several measures of autonomic impairment that are known ...

The 4-year progression-free survival rate was 69% and the overall survival rate was 80% for all patients. A short induction regimen followed by thiotepa-busulfan-based autologous stem cell ...

The new study will investigate safety and pharmacokinetic parameters to guide KRRO-110 dose selection in future stage studies. The Australian Bellberry Human Research Ethics Committee (HREC) and the ...

All patients showed evidence of fatty infiltration of the left ventricle, although to varying degrees. Diffuse fibrofatty replacement of myocardial tissue may begin to occur earlier than previously ...

Transcranial direct current stimulation, alongside aerobic exercise, may improve fatigue in patients with GPA. A patient with granulomatosis with polyangiitis (GPA)—a form of antineutrophil ...

Patients with mastocytosis mostly had similar rates of atopy and allergies as the general population, except for certain conditions such as asthma. While patients with mastocytosis (both systemic [SM] ...