A Latin American study of vosoritide in achondroplasia showed that height Z-scores rose from 0.33 at 6 months to 1.58 at 36 ...

Some baseline characteristics are suggestive of more advanced disease in women with ATTR than men, according to data ...

Valbenazine, a VMAT2 inhibitor approved for chorea in HD, led to improved quality of life in the KINECT-PRO study in tardive ...

In the ALPHA trial, add-on danicopan improved hematologic parameters in patients with PNH with and without bone marrow ...

A case was reported in which FSGS preceded the classic manifestations of anti-Jo-1-positive antisynthetase syndrome.

Urine albumin-to-protein ratio varied across 3 IgA nephropathy phenotypes and accompanied distinct podocyte and glomerular ...

In a retrospective study, patients with MG who received a cycle of efgartigimod experienced more rapid improvement than those ...

A woman living with neuromyelitis optica spectrum disorder (NMOSD) struggles to discern which symptoms signal a medical ...

Determining what antigens the fetus carries can help clinicians monitor pregnancies that are genuinely at risk of HDFN and ...

Early findings from a natural history study of DS and other SCN1A-related epilepsies revealed a range of educational and ...

Emerging targeted and steroid-sparing treatment strategies for ANCA-associated vasculitis are changing the landscape of ...

Anti-D microtitration may differentiate antibodies from an RhIg injection and antibodies due to maternal alloimmunization ...