The Pharmacokinetic Profile of Palovarotene: An Open-Label Phase I Trial Investigating the Effect of Food and Potential for Drug–Drug Interaction in Healthy Participants

por Marino, Rose, Dube, Louise, Ogier, Julien, Le Quan Sang, Kim-Hanh
Publicado 2023

A Pharmacokinetic, Safety, and Tolerability Trial of Palovarotene in Healthy Japanese and Non-Japanese Participants

por Dube, Louise, Haga, Nobuhiko, Grogan, Donna, Ogier, Julien, Le Quan Sang, Kim-Hanh
Publicado 2023

Prevalence of fibrodysplasia ossificans progressiva (FOP) in France: an estimate based on a record linkage of two national databases

por Baujat, Geneviève, Choquet, Rémy, Bouée, Stéphane, Jeanbat, Viviane, Courouve, Laurène, Ruel, Amélie, Michot, Caroline, Le Quan Sang, Kim-Hanh, Lapidus, David, Messiaen, Claude, Landais, Paul, Cormier-Daire, Valérie
Publicado 2017

Study methodology and insights from the palovarotene clinical development program in fibrodysplasia ossificans progressiva

por Pignolo, Robert J., Al Mukaddam, Mona, Baujat, Geneviève, Brown, Matthew A., De Cunto, Carmen, Hsiao, Edward C., Keen, Richard, Le Quan Sang, Kim-Hanh, Grogan, Donna R., Marino, Rose, Strahs, Andrew R., Kaplan, Frederick S.
Publicado 2023

Patients with ACVR1(R206H) mutations have an increased prevalence of cardiac conduction abnormalities on electrocardiogram in a natural history study of Fibrodysplasia Ossificans P...

por Kou, Samuel, De Cunto, Carmen, Baujat, Geneviève, Wentworth, Kelly L., Grogan, Donna R., Brown, Matthew A., Di Rocco, Maja, Keen, Richard, Al Mukaddam, Mona, le Quan Sang, Kim-Hanh, Masharani, Umesh, Kaplan, Frederick S., Pignolo, Robert J., Hsiao, Edward C.
Publicado 2020

Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study

por Jones, Simon A., Rojas-Caro, Sandra, Quinn, Anthony G., Friedman, Mark, Marulkar, Sachin, Ezgu, Fatih, Zaki, Osama, Gargus, J. Jay, Hughes, Joanne, Plantaz, Dominique, Vara, Roshni, Eckert, Stephen, Arnoux, Jean-Baptiste, Brassier, Anais, Le Quan Sang, Kim-Hanh, Valayannopoulos, Vassili
Publicado 2017

Effects of Nitisinone on Oxidative and Inflammatory Markers in Alkaptonuria: Results from SONIA1 and SONIA2 Studies

por Braconi, Daniela, Geminiani, Michela, Psarelli, Eftychia Eirini, Giustarini, Daniela, Marzocchi, Barbara, Rossi, Ranieri, Bernardini, Giulia, Spiga, Ottavia, Gallagher, James A., Le Quan Sang, Kim-Hanh, Arnoux, Jean-Baptiste, Imrich, Richard, Al-Sbou, Mohammed S., Gornall, Matthew, Jackson, Richard, Ranganath, Lakshminarayan R., Santucci, Annalisa
Publicado 2022

Prevalence of rare mitochondrial DNA mutations in mitochondrial disorders

por Bannwarth, Sylvie, Procaccio, Vincent, Lebre, Anne Sophie, Jardel, Claude, Chaussenot, Annabelle, Hoarau, Claire, Maoulida, Hassani, Charrier, Nathanaël, Gai, Xiaowu, Xie, Hongbo M, Ferre, Marc, Fragaki, Konstantina, Hardy, Gaëlle, Mousson de Camaret, Bénédicte, Marlin, Sandrine, Dhaenens, Claire Marie, Slama, Abdelhamid, Rocher, Christophe, Paul Bonnefont, Jean, Rötig, Agnès, Aoutil, Nadia, Gilleron, Mylène, Desquiret-Dumas, Valérie, Reynier, Pascal, Ceresuela, Jennifer, Jonard, Laurence, Devos, Aurore, Espil-Taris, Caroline, Martinez, Delphine, Gaignard, Pauline, Le Quan Sang, Kim-Hanh, Amati-Bonneau, Patrizia, Falk, Marni J, Florentz, Catherine, Chabrol, Brigitte, Durand-Zaleski, Isabelle, Paquis-Flucklinger, Véronique
Publicado 2013