title: Proprotein Convertase Subtilisin Kexin Type 9 Inhibition for Autosomal Recessive Hypercholesterolemia-Brief Report.
abstract: Proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitors lower low-density lipoprotein (LDL) cholesterol in the vast majority of patients with autosomal dominant familial hypercholesterolemia. Will PCSK9 inhibition with monoclonal antibodies, in particular alirocumab, be of therapeutic value for patients with autosomal recessive hypercholesterolemia (ARH)?
  
  
  
  Primary lymphocytes were obtained from 28 genetically characterized ARH patients and 11 controls. ARH lymphocytes treated with mevastatin were incubated with increasing doses of recombinant PCSK9 with or without saturating concentrations of alirocumab. Cell surface LDL receptor expression measured by flow cytometry and confocal microscopy was higher in ARH than in control lymphocytes. PCSK9 significantly reduced LDL receptor expression in ARH lymphocytes albeit to a lower extent than in control lymphocytes (25% versus 76%, respectively), an effect reversed by alirocumab. Fluorescent LDL cellular uptake, also measured by flow cytometry, was reduced in ARH lymphocytes compared with control lymphocytes. PCSK9 significantly lowered LDL cellular uptake in ARH lymphocytes, on average by 18%, compared with a 46% reduction observed in control lymphocytes, an effect also reversed by alirocumab. Overall, the effects of recombinant PCSK9, and hence of alirocumab, on LDL receptor expression and function were significantly less pronounced in ARH than in control cells.
  
  
  
  PCSK9 inhibition with alirocumab on top of statin treatment has the potential to lower LDL cholesterol in some autosomal recessive hypercholesterolemia patients.
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Aurélie
authors.initials: A
authors.lastname: Thedrez
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Barbara
authors.initials: B
authors.lastname: Sjouke
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Maxime
authors.initials: M
authors.lastname: Passard
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Simon
authors.initials: S
authors.lastname: Prampart-Fauvet
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Alexis
authors.initials: A
authors.lastname: Guédon
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Mikael
authors.initials: M
authors.lastname: Croyal
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Geesje
authors.initials: G
authors.lastname: Dallinga-Thie
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Jorge
authors.initials: J
authors.lastname: Peter
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Dirk
authors.initials: D
authors.lastname: Blom
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Milco
authors.initials: M
authors.lastname: Ciccarese
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Angelo B
authors.initials: AB
authors.lastname: Cefalù
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Livia
authors.initials: L
authors.lastname: Pisciotta
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Raul D
authors.initials: RD
authors.lastname: Santos
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Maurizio
authors.initials: M
authors.lastname: Averna
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Frederick
authors.initials: F
authors.lastname: Raal
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Paolo
authors.initials: P
authors.lastname: Pintus
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Maria
authors.initials: M
authors.lastname: Cossu
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Kees
authors.initials: K
authors.lastname: Hovingh
authors.affiliation: From the Inra UMR 1280, Université de Nantes, Faculté de Médecine, Nantes, France (A.T., M.P., S.P.-F., A.G., M.C., G.L.); Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands (B.S., G.D.-T., J.P., K.H.); Lipidology Division of Internal Medicine, University of Cape Town, Cape Town, South Africa (D.B.); Dipartimiento di Nefrologia Dialisi e Trapianto, SS Annunziata Hospital, Sassari, Italy (M.C., M.C.); University of Palermo, School of Medicine, Palermo, Italy (A.B.C., M.A.); University of Genoa, Genoa, Italy (L.P.); Lipid Clinic Heart Institute (InCor) University of Sao Paulo Medica School Hospital, Sao Paulo, Brazil (R.D.S.); Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.R.); Dipartimento di Medicina Interna, Brotzu Hospital, Cagliari, Italy (P.P.); Inserm UMR 1188, Sainte Clotilde, France (G.L.); Université de la Réunion, Faculté de Médecine, Saint Denis de la Réunion, France (G.L.); and CHU de la Réunion, Saint-Denis de la Réunion, France (G.L.).
authors.forename: Gilles
authors.initials: G
authors.lastname: Lambert
mesh_terms: D048868:Adaptor Proteins, Signal Transducing / Q000235:genetics; D000293:Adolescent; D000328:Adult; D000911:Antibodies, Monoclonal / Q000494:pharmacology*; D061067:Antibodies, Monoclonal, Humanized; D000924:Anticholesteremic Agents / Q000494:pharmacology*; D016022:Case-Control Studies; D002478:Cells, Cultured; D008078:Cholesterol, LDL / Q000378:metabolism; D004359:Drug Therapy, Combination; D005260:Female; D020022:Genetic Predisposition to Disease; D006801:Humans; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors / Q000494:pharmacology; D006937:Hypercholesterolemia / Q000175:diagnosis / Q000188:drug therapy* / Q000201:enzymology / Q000235:genetics; D008148:Lovastatin / Q000031:analogs & derivatives / Q000494:pharmacology; D008214:Lymphocytes / Q000187:drug effects* / Q000201:enzymology; D008297:Male; D008875:Middle Aged; D009154:Mutation; D000091362:PCSK9 Inhibitors; D010641:Phenotype; D000071449:Proprotein Convertase 9 / Q000378:metabolism; D011973:Receptors, LDL / Q000378:metabolism; D015842:Serine Proteinase Inhibitors / Q000494:pharmacology*; D055815:Young Adult; D006952:Hyperlipoproteinemia Type III
chemical_list: D048868:Adaptor Proteins, Signal Transducing; D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D000924:Anticholesteremic Agents; D008078:Cholesterol, LDL; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors; C573412:LDLR protein, human; C469745:LDLRAP1 protein, human; D000091362:PCSK9 Inhibitors; D011973:Receptors, LDL; D015842:Serine Proteinase Inhibitors; C012258:mevastatin; D008148:Lovastatin; C472125:PCSK9 protein, human; D000071449:Proprotein Convertase 9; C571059:alirocumab
keywords: alirocumab; hypercholesterolemia; proprotein convertase subtilisin kexin type 9; receptors, LDL; therapeutics