Rare Disease Advisor

Maintenance HMAs May Improve Outcomes After AlloHCT in MDS

Hypomethylating agent maintenance after allogeneic HCT improved relapse-free and overall survival in myelodysplastic ...
Rare Disease Advisor

Venetoclax Delivers Durable Survival in High-Risk B-Cell ALL

Venetoclax-based therapy yielded 93.3% overall survival at 36 months in high-risk B-cell acute lymphoblastic leukemia (ALL).
Rare Disease Advisor

Neuroimaging in Siblings With Juvenile HD Reveals Striatal Atrophy, Possible Spinal Involvement

Neuroimaging findings in 3 siblings with juvenile HD raised the possibility that spinal involvement may be a feature of the ...
Rare Disease Advisor

Targeting Apolipoprotein CIII in FCS and Beyond

Two new FCS therapies offer potent triglyceride lowering, but further studies are needed to confirm the promise shown in ...
Rare Disease Advisor

Tremor Is Common in CIDP but Not Always Perceptible

Tremor and myoclonus may contribute to disability in patients with CIDP, although certain therapies may reduce symptoms.
Rare Disease Advisor

Researchers Identify Factor That May Link Huntington Disease Progression and Neuronal Aging

In a new study, age-associated NFKB1 upregulation increased neuronal vulnerability in samples from patients with Huntington ...
Rare Disease Advisor

Ofirnoflast Sustains Transfusion Independence in Lower-Risk MDS

Ofirnoflast produced erythroid-specific hematologic improvement in 63% of efficacy-evaluable patients with lower-risk MDS.
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Contrast-Enhanced Ultrasonography Detects Altered Muscle Perfusion in PMDs Such as TK2d

Pilot study results demonstrate the feasibility of contrast-enhanced ultrasonography in evaluating muscle perfusion in ...
Rare Disease Advisor

Intrauterine Transfusions Using ABO-Matched Rh-Negative Blood

Intrauterine blood transfusions, used to treat conditions such as FNAIT, may sometimes use the fetus’s ABO-compatible ...
Rare Disease Advisor

FDA Approves Apitegromab, First Muscle-Targeting Therapy for Children and Adults With SMA

The FDA approved apitegromab, making it the first and only therapy for patients with SMA that directly targets muscle ...
Rare Disease Advisor

Understanding and Managing Cardiovascular Risk in Patients With MDS

Standard risk calculators may underestimate cardiovascular disease risk in patients with hematologic malignancies. Cardiovascular disease (CVD) affects a substantial portion of patients with ...
Rare Disease Advisor

Inflammatory Cytokine Pattern Emerges in Fibrotic Low-Risk MDS

Low-risk myelodysplastic syndromes (MDS) with substantial marrow fibrosis exhibited an inflammatory serum profile involving ...