Rare Disease Advisor

Preemptive Donor Lymphocyte Infusion May Reduce Relapse After HSCT in MDS

Preemptive donor lymphocyte infusion (DLI) before HSCT yields superior outcomes compared with therapeutic DLI in patients with MDS.
Rare Disease Advisor

EMA’s CHMP Backs Olezarsen for Severe Hypertriglyceridemia Beyond FCS

The CHMP recommended extending the indications for olezarsen in the EU to causes of severe hypertriglyceridemia other than ...
Rare Disease Advisor

Chronic Kidney Disease Raises the Risk of Myelodysplastic Syndromes

Chronic kidney disease (CKD) stages G3 and G4 were associated with a 57% higher subdistribution hazard of incident MDS in a ...
Rare Disease Advisor

Malignancy Leads Causes of Death in Brazilian Type 1 Gaucher Disease Cohort

In a Brazilian study, cancer was the most common cause of death in adults with GD type 1, who also had a notable comorbidity ...
Rare Disease Advisor

Fenebrutinib Curbs Chronic Active Lesions in Relapsing and Progressive MS: Pooled Phase 3 Data

Pooled phase 3 trial data showed that fenebrutinib slowed the growth of slowly expanding lesions in both RMS and PPMS.
Rare Disease Advisor

Poor Survival Identified in Older, Transplant-Ineligible Patients With MCL

Older, transplant-ineligible patients with MCL experienced poor survival, with median OS declining from first-line to ...
Rare Disease Advisor

How My CIDP Prepared Me to Face NMOSD

It might sound odd, but I’m grateful I was already ill when I experienced my first neuromyelitis optica spectrum disorder ...