- 专利标题: METHODS FOR DETERMINING WHETHER A PATIENT SUFFERING FROM RHABDOMYOLYSIS ACHIEVES A RESPONSE WITH A TLR9 ANTAGONIST
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申请号: US16633912申请日: 2018-07-26
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公开(公告)号: US20210087633A1公开(公告)日: 2021-03-25
- 发明人: Pascale DE LONLAY-DEBENEY , Peter VAN ENDERT , Marine MADRANGE , Yamina HAMEL , François-Xavier MAUVAIS
- 申请人: INSERM (INSTITUTE NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , FONDATION IMAGINE , UNIVERSITÉ DE PARIS , ASSISTANCE PUBLIQUE-HÔPITAUX DE PARIS (APHP) , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS)
- 申请人地址: FR Paris; FR Paris; FR Paris; FR Paris; FR Paris
- 专利权人: INSERM (INSTITUTE NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE),FONDATION IMAGINE,UNIVERSITÉ DE PARIS,ASSISTANCE PUBLIQUE-HÔPITAUX DE PARIS (APHP),CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS)
- 当前专利权人: INSERM (INSTITUTE NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE),FONDATION IMAGINE,UNIVERSITÉ DE PARIS,ASSISTANCE PUBLIQUE-HÔPITAUX DE PARIS (APHP),CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS)
- 当前专利权人地址: FR Paris; FR Paris; FR Paris; FR Paris; FR Paris
- 优先权: EP17306007.0 20170727
- 国际申请: PCT/EP2018/070256 WO 20180726
- 主分类号: C12Q1/6883
- IPC分类号: C12Q1/6883 ; C12Q1/686
摘要:
The inventors initially participated to the identification of LPIN1 mutations as a cause for massive rhabdomyolysis episodes in children, triggered by febrile illness. The inventors have suggested that TLR9 antagonists would be suitable for the treatment of rhabdomyolysis (WO2017085115). The inventors thus treated 2 patients with lipin-1 disease by a TRL9 antagonist (hydroxychloroquine). They showed that the accumulation of mtDNA in plasma of the two patients before treatment decreases under treatment. When the treatment was stopped, the accumulation of mtDNA reappeared, then normalized when treatment was resumed. Accordingly, the present invention relates to a method for determining whether a patient suffering from rhabdomyolysis achieves a response with a TLR9 antagonist comprising determining the amount of mitochondrial DNA (mtDNA) in a blood sample obtained from the patient (e.g. by PCR).
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