J. M. Campistol


AuthID: R-00F-FWR

Publications Awaiting Confirmation

1
TITLE: The Interplay between Pathophysiological Pathways in Early-Onset Severe Preeclampsia Unveiled by Metabolomics  Full Text
AUTHORS: Youssef, L; Crovetto, F; Simoes, RV; Miranda, J; Paules, C; Blasco, M; Palomo, M; Garcia Caldero, H; Tura Ceide, O; Dantas, AP; Hernandez Gea, V; Herrero, P; Canela, N; Campistol, JM; Garcia Pagan, JC; Diaz Ricart, M; Gratacos, E; Crispi, F;
PUBLISHED: 2022, SOURCE: LIFE-BASEL, VOLUME: 12, ISSUE: 1
INDEXED IN: Scopus WOS
2
TITLE: Defining tetrahydrobiopterin responsiveness in phenylketonuria: Survey results from 38 countries  Full Text
AUTHORS: Evers, RAF; van Wegberg, AMJ; Ahring, K; Beblo, S; Belanger Quintana, A; Bosch, AM; Burlina, A; Campistol, J; Coskun, T; Feillet, F; Gizewska, M; Huijbregts, SCJ; Kearney, S; Langeveld, M; Leuzzi, V; Maillot, F; Muntau, AC; Rocha, JC; Romani, C; Trefz, FK; MacDonald, A; van Spronsen, FJ; ...More
PUBLISHED: 2021, SOURCE: MOLECULAR GENETICS AND METABOLISM, VOLUME: 132, ISSUE: 4
INDEXED IN: Scopus WOS
3
TITLE: PKU dietary handbook to accompany PKU guidelines  Full Text
AUTHORS: MacDonald, A; Van Wegberg, AMJ; Ahring, K; Beblo, S; Bélanger Quintana, A; Burlina, A; Campistol, J; Coşkun, T; Feillet, F; Giżewska, M; Huijbregts, SC; Leuzzi, V; Maillot, F; Muntau, AC; Rocha, JC; Rocha, JC; Romani, C; Trefz, F; Van Spronsen, FJ;
PUBLISHED: 2020, SOURCE: ORPHANET JOURNAL OF RARE DISEASES, VOLUME: 15, ISSUE: 1
INDEXED IN: Scopus WOS
4
TITLE: A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis  Full Text
AUTHORS: Teresa Coelho; David Adams; Isabel Conceicao; Marcia Waddington Cruz; Hartmut H Schmidt; Juan Buades; Josep Campistol; John L Berk; Michael Polydefkis; Jing Jing Wang; Jihong H Chen; Marianne T Sweetser; Jared Gollob; Ole B Suhr;
PUBLISHED: 2020, SOURCE: ORPHANET JOURNAL OF RARE DISEASES, VOLUME: 15, ISSUE: 1
INDEXED IN: Scopus WOS
5
TITLE: PKU dietary handbook to accompany PKU guidelines (vol 15, 171, 2020)  Full Text
AUTHORS: MacDonald, A; van Wegberg, AMJ; Ahring, K; Beblo, S; Belanger Quintana, A; Burlina, A; Campistol, J; Coskun, T; Feillet, F; Gizewska, M; Huijbregts, SC; Leuzzi, V; Maillot, F; Muntau, AC; Rocha, JC; Romani, C; Trefz, F; van Spronsen, FJ;
PUBLISHED: 2020, SOURCE: ORPHANET JOURNAL OF RARE DISEASES, VOLUME: 15, ISSUE: 1
INDEXED IN: WOS
6
TITLE: Cardiac phenotype in ATP1A3-related syndromes A multicenter cohort study
AUTHORS: Balestrini, S; Mikati, MA; Alvarez Garcia Roves, R; Carboni, M; Hunanyan, AS; Kherallah, B; McLean, M; Prange, L; De Grandis, E; Gagliardi, A; Pisciotta, L; Stagnaro, M; Veneselli, E; Campistol, J; Fons, C; Pias Peleteiro, L; Brashear, A; Miller, C; Samoes, R; Brankovic, V; Padiath, QS; Potic, A; Pilch, J; Vezyroglou, A; Bye, AME; Davis, AM; Ryan, MM; Semsarian, C; Hollingsworth, G; Scheffer, IE; Granata, T; Nardocci, N; Ragona, F; Arzimanoglou, A; Panagiotakaki, E; Carrilho, I; Zucca, C; Novy, J; Parowicz, M; Weckhuysen, S; Pons, R; Groppa, S; Sinden, DS; Pitt, GS; Tinker, A; Ashworth, M; Michalak, Z; Thom, M; Cross, JH; Vavassori, R; Kaski, JP; Sisodiya, SM; Dzieiyc, K; Mazurkiewicz Beldzinska, M; ...More
PUBLISHED: 2020, SOURCE: NEUROLOGY, VOLUME: 95, ISSUE: 21
INDEXED IN: WOS
7
TITLE: Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
AUTHORS: Adams, D; Gonzalez Duarte, A; O'Riordan, WD; Yang, CC; Ueda, M; Kristen, AV; Tournev, I; Schmidt, HH; Coelho, T; Berk, JL; Lin, KP; Vita, G; Attarian, S; Plante Bordeneuve, V; Mezei, MM; Campistol, JM; Buades, J; Brannagan, TH; Kim, BJ; Oh, J; Parman, Y; Sekijima, Y; Hawkins, PN; Solomon, SD; Polydefkis, M; Dyck, PJ; Gandhi, PJ; Goyal, S; Chen, J; Strahs, AL; Nochur, SV; Sweetser, MT; Garg, PP; Vaishnaw, AK; Gollob, JA; Suhr, OB; ...More
PUBLISHED: 2018, SOURCE: NEW ENGLAND JOURNAL OF MEDICINE, VOLUME: 379, ISSUE: 1
INDEXED IN: Scopus WOS
8
TITLE: Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
AUTHORS: Benson, MD; Waddington Cruz, M; Berk, JL; Polydefkis, M; Dyck, PJ; Wang, AK; Plante Bordeneuve, V; Barroso, FA; Merlini, G; Obici, L; Scheinberg, M; Brannagan, TH; Litchy, WJ; Whelan, C; Drachman, BM; Adams, D; Heitner, SB; Conceicao, I; Schmidt, HH; Vita, G; Campistol, JM; Gamez, J; Gorevic, PD; Gane, E; Shah, AM; Solomon, SD; Monia, BP; Hughes, SG; Kwoh, TJ; McEvoy, BW; Jung, SW; Baker, BF; Ackermann, EJ; Gertz, MA; Coelho, T; ...More
PUBLISHED: 2018, SOURCE: NEW ENGLAND JOURNAL OF MEDICINE, VOLUME: 379, ISSUE: 1
INDEXED IN: Scopus WOS
9
TITLE: Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study  Full Text
AUTHORS: Ole B Suhr; Teresa Coelho; Juan Buades; Jean Pouget; Isabel Conceicao; John Berk; Hartmut Schmidt; Marcia Waddington Cruz; Josep M Campistol; Brian R Bettencourt; Akshay Vaishnaw; Jared Gollob; David Adams;
PUBLISHED: 2015, SOURCE: ORPHANET JOURNAL OF RARE DISEASES, VOLUME: 10, ISSUE: 1
INDEXED IN: Scopus WOS CrossRef
10
TITLE: PHASE 2 OPEN-LABEL EXTENSION STUDY OF PATISIRAN, AN INVESTIGATIONAL RNAI THERAPEUTIC FOR THE TREATMENT OF FAMILIAL AMYLOID POLYNEUROPATHY
AUTHORS: David Adams; Ole Suhr; Isabel Conceicao; Marcia Waddington Cruz; Hartmut Schmidt; Juan Buades; Josep Campistol; Jean Pouget; John Berk; Teresa Coelho;
PUBLISHED: 2015, SOURCE: Annual Meeting of the Association-of-British-Neurologists (ABN) in JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY, VOLUME: 86, ISSUE: 11
INDEXED IN: WOS CrossRef
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