Valbenazine, a VMAT2 inhibitor approved for chorea in HD, led to improved quality of life in the KINECT-PRO study in tardive ...
Determining what antigens the fetus carries can help clinicians monitor pregnancies that are genuinely at risk of HDFN and ...
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 ...
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 ...
A case was reported in which FSGS preceded the classic manifestations of anti-Jo-1-positive antisynthetase syndrome.
Some baseline characteristics are suggestive of more advanced disease in women with ATTR than men, according to data ...
In the ALPHA trial, add-on danicopan improved hematologic parameters in patients with PNH with and without bone marrow ...
IDH inhibitors produced CRi in 55.5% of children with relapsed or refractory IDH-mutated AML or myelodysplastic syndromes ...
Early findings from a natural history study of DS and other SCN1A-related epilepsies revealed a range of educational and ...
Long-term prophylaxis with lanadelumab was associated with durable reductions in HAE attack rates and lasting improvements in ...
Urine albumin-to-protein ratio varied across 3 IgA nephropathy phenotypes and accompanied distinct podocyte and glomerular ...
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