Gene/Protein
Disease
Symptom
Drug
Enzyme
Compound
Pivot Concepts:
Gene/Protein
Disease
Symptom
Drug
Enzyme
Compound
Target Concepts:
Gene/Protein
Disease
Symptom
Drug
Enzyme
Compound
Query: UMLS:C0268596 (
EMA
)
2,520
document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)
Approximately 50% of
aHUS
patients have an underlying inherited and/or acquired abnormality of complement which predisposes to excessive activation of the alternative pathway. Use of complement inhibitors such as eculizumab to treat
aHUS
is therefore logical. Anecdotal reports and subsequent open-label trials demonstrated the efficacy of eculizumab in
aHUS
leading to approval by both the FDA and
EMA
. NHS England established in 2013 an interim national service for
aHUS
including funding for eculizumab for both new patients and those undergoing transplantation. NICE guidance now also recommends eculizumab for funding within the NHS in England under the coordination of an expert centre. The investigation and response to treatment in this cohort provides a unique resource for patient stratification.
...
PMID:Patient stratification and therapy in atypical haemolytic uraemic syndrome (aHUS). 2603 15
