Treatment with antenatal IVIG and steroids protected a second sibling from developing the bleeding symptoms and other ...
Aarush approached Veer with a novel concept: using AI to create a public, searchable free platform built exclusively for rare diseases.
Increased emphasis has focused on parent-led and other nonpharmacologic approaches intended to support the well-being of ...
Deutetrabenazine significantly reduced chorea severity over 16 weeks in a prospective study of Chinese adults with Huntington disease (HD).
The earlier onset of PPF is associated with a higher risk of mortality in patients with CTD-ILD, according to a recent study.
Even when a treatment plan works to prevent NMOSD relapses, the regimen can take a mental and physical toll on the patient.
Patients with IPF treated with inhaled treprostinil at 9 breaths per session or more showed greater improvement in forced ...
Venetoclax improved response rates in TP53-mutant MDS, particularly among patients with higher marrow blast percentages.
MRD can reveal residual MDS beyond morphology, but heterogeneous clones and assay limitations complicate interpretation.
An SLC25A51 VUS found in 2 siblings was associated with proteomic changes involving mitochondrial metabolism and cytoskeletal ...
At year 2 of the HOPE-3 OLE, patients with DMD experienced a 76% reduction in the rate of upper-limb decline after initiating ...
The results of a cell study showed how distinct responses to mitochondrial stress may contribute to phenotypic variability ...
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