Two new FCS therapies offer potent triglyceride lowering, but further studies are needed to confirm the promise shown in ...
Neuroimaging findings in 3 siblings with juvenile HD raised the possibility that spinal involvement may be a feature of the ...
Tremor and myoclonus may contribute to disability in patients with CIDP, although certain therapies may reduce symptoms.
Venetoclax-based therapy yielded 93.3% overall survival at 36 months in high-risk B-cell acute lymphoblastic leukemia (ALL).
Hypomethylating agent maintenance after allogeneic HCT improved relapse-free and overall survival in myelodysplastic ...
Ofirnoflast produced erythroid-specific hematologic improvement in 63% of efficacy-evaluable patients with lower-risk MDS.
In a new study, age-associated NFKB1 upregulation increased neuronal vulnerability in samples from patients with Huntington ...
The FDA approved apitegromab, making it the first and only therapy for patients with SMA that directly targets muscle ...
Intrauterine blood transfusions, used to treat conditions such as FNAIT, may sometimes use the fetus’s ABO-compatible ...
Follow-up testing after atypical cell-free DNA (cfDNA) screening, sometimes used in FNAIT assessment, may identify genetic ...
Pilot study results demonstrate the feasibility of contrast-enhanced ultrasonography in evaluating muscle perfusion in ...
Many issues can cause maternal anxiety and stress, including a rare disease diagnosis such as fetal and neonatal alloimmune ...
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