Abstract: Lipin-1 deficiency is a rare, life-threatening condition that causes severe rhabdomyolysis episodes (RM) triggered by febrile illness and effort. Now, the inventors treated 10 patients with LPIN1 mutations with hydroxychloroquine (HCQ) in an off open-label use phases 1 and 2 study, to assess safety, clinical, and biological effects of the drug. A first inclusion group of patients were treated with oral HCQ at a dose of 6.5 mg/Kg/day in one intake, not exceeding 400 mg/day. Five patients have not presented any new acute RM under treatment, except for 2 patients experimented one and two episodes of RM respectively despite HCQ in a context of gastroenteritis. Plasma levels of HCQ were in the range of 400 ng/ml except in the two patients who experimented RM, in whom the plasma HCQ levels were higher (1000 ng/ml). With a therapeutic adjustment, in order to maintain plasma levels of HCQ under 700 ng/ml, in a new group of patients, two patients did not suffer from new acute RM under treatment.

Abstract: 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).

Abstract: The present invention is directed to 5-membered heteroaryl compounds containing a hydroxamate moiety of Formula I, pharmaceutically acceptable salts or solvates thereof, and their use as sensitizers for chemotherapy of malignant tumors.

Abstract: The present invention relates to nanoparticles, kits, methods and compositions which are suitable for increasing the number of B regulatory (Breg) cells in a population of B cells; for producing Interleukin-10 (IL-10) or TGF-?. The inventors have shown that biocompatible nanoparticles comprising an antigen may be used for inducing B regulatory (Breg) cells. This production, either ex vivo, or in vivo, or in vitro, was associated to temporary or lasting remission of disease in spontaneously diabetic NOD mice.

Abstract: The present invention relates to nanoparticles, methods and compositions which are suitable for the detection and/or follow-up and/or treatment of type 1 diabetes. In particular, it relates to biocompatible tolerogenic nanoparticles comprising at least: (i) a ligand which can bind to an aryl hydrocarbon receptor (AHR) transcription factor; an (ii) a diabetes autoantigen selected from: insulin, preproinsulin, proinsulin, or an immunologically active fragment thereof The inventors have shown that such biocompatible tolerogenic nanoparticles are efficient for the identification of type-1 diabetes. It has also been shown that they can accumulate into the pancreas, and induce temporary or lasting remission of disease in spontaneously diabetic NOD mice. Kits and compositions are further provided.

Abstract: 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).

Abstract: The present invention relates to methods and pharmaceutical compositions for treating rhabdomyolysis.

Abstract: The present invention relates to methods and pharmaceutical compositions for treating rhabdomyolysis.

Abstract: The invention is related to fusion proteins comprising two or more IgG binding domains of streptococcal protein G, linked to a polypeptide of interest. These fusion proteins can be complexed with IgG antibodies directed against surface receptors of target cells, allowing their delivery to said target cells.

Abstract: the invention concerns autoreactive peptides, peptide MHC complexes, t cell subpopulations which react therewith as well as diagnostic and therapeutic applications of these compounds.