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7. Identification of a new factor involved in DNA methylation-mediated repression of latent HIV-1

Author

Bouchat, S., Verdikt, R., Delacourt, N., Vanhulle, C., Driessche, B., Darcis, G., Pasternak, A., Véronique Avettand-Fenoel, Ledouce, V., Bendoumou, M., Schwartz, C., Wit, S., Berkhout, B., Gautier, V., Rouzioux, C., Rohr, O., and Lint, C.

Subjects
0301 basic medicine, Genetics, Epidemiology, Immunology, Public Health, Environmental and Occupational Health, Human immunodeficiency virus (HIV), Biology, medicine.disease_cause, Microbiology, QR1-502, 03 medical and health sciences, 030104 developmental biology, Infectious Diseases, Virology, DNA methylation, medicine, Identification (biology), Public aspects of medicine, RA1-1270, Psychological repression
Published
2017

9. Identification of a new factor involved in DNA methylation-mediated repression of latent HIV-1

Author

Van Lint, C., primary, Bouchat, S., additional, Verdikt, R., additional, Van Driessche, B., additional, Pasternak, A., additional, Darcis, G., additional, Delacourt, N., additional, Vanhulle, C., additional, Avettand-fenoel, V., additional, Ledouce, V., additional, Schwartz, C., additional, Necsoi, C., additional, De Wit, S., additional, Berkhout, B., additional, Gautier, V., additional, Rouzioux, C., additional, and Rohr, O., additional

Published
2017
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10. O2 Identification of a new factor involved in DNA methylation-mediated repression of latent HIV-1

Author

Bouchat, S., primary, Verdikt, R., additional, Delacourt, N., additional, Vanhulle, C., additional, Van Driessche, B., additional, Darcis, G., additional, Pasternak, A., additional, Avettand-Fenoel, V., additional, Necsoi, C., additional, Ledouce, V., additional, Bendoumou, M., additional, Schwartz, C., additional, De Wit, S., additional, Saez-Cirion, A., additional, Berkhout, B., additional, Gautier, V., additional, Rouzioux, C., additional, Rohr, O., additional, and Van Lint, C., additional

Published
2017
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13. Gain of function mutations in IFIH1 cause a spectrum of human disease phenotypes associated with upregulated type I interferon signaling

Author

Rice, Gi, Del Toro Duany, Y, Jenkinson, Em, Forte, Gm, Anderson, Bh, Ariaudo, G, Bader-Meunier, B, Baildm, Em, Battini, R, Beresford, Mw, Casarano, M, Chouchane, M, Cimaz, R, Collins, Ae, Cordeiro, Nj, Dale, Rc, Davidson, Je, Waelel, De, Desguerre, I, Faivre, L, Fazzi, E, Isidor, B, Lagae, L, Larchman, Ar, Lebon, P, Li, C, Livingston, Jh, Lourenço, Cm, Mancardi, Mm, Masurel-Paulet, A, Mcinnes, Ib, Menezes, Mp, Mignot, C, O'Sullivan, J, Orcesi, S, Picco, Pp, Riva, E, Robinson, Ra, Rodriguez, D, Salvatici, E, Scott, C, Szybowska, M, Tolmie, Jl, Vanderver, A, Vanhulle, C, Vieira, Jp, Webb, K, Whitney, Rn, Williams, Sg, Wolfe, La, Zuberi, Sm, Hur, S, and Crow, Yj

Published
2014

14. Non-ambulant duchenne patients theoretically treatable by exon 53 skipping have severe phenotype

Author

Servais, L, Montus, M, Le Guiner, C, Ben Yaou, R, Annoussamya, M, Moraux, A, Hogrel, J-Y, Seferian, A M, Zehrouni, K, Lo Moing, A-G, Gidaro, T, Vanhulle, C, Laugel, V, Butoianu, N, Cuisset, J-M, Sabouraud, P, Cances, C, Klein, A, Leturcq, F, Moullier, P, Voit, T, Servais, L, Montus, M, Le Guiner, C, Ben Yaou, R, Annoussamya, M, Moraux, A, Hogrel, J-Y, Seferian, A M, Zehrouni, K, Lo Moing, A-G, Gidaro, T, Vanhulle, C, Laugel, V, Butoianu, N, Cuisset, J-M, Sabouraud, P, Cances, C, Klein, A, Leturcq, F, Moullier, P, and Voit, T

Abstract

Background: Exon skipping therapy is an emerging approach in Duchenne Muscular Dystrophy (DMD). Antisense oligonucleotides that induce skipping of exon 51, 44, 45, or 53 are currently being evaluated in clinical trials. These trials were designed on the basis of data available in general DMD population. Objectives: Our objective was to compare the clinical and functional statuses of non-ambulant DMD patients theoretically treatable by exon 53 skipping and of DMD patients with other mutations. Methods: We first compared fifteen non-ambulant DMD patients carrying deletions theoretically treatable by exon 53 skipping (DMD-53) with fifteen closely age-matched DMD patients with mutations not treatable by exon 53 skipping (DMD-all-non-53) then with fifteen DMD patients carrying deletions not treatable by exon 53 skipping (DMD-del-non-53). Results: We found that DMD-53 patients had a lower left ventricular ejection fraction, more contractures and they tend to have weaker grips and pinch strengths than other DMD patients. DMD-53 patients lost ambulation significantly younger than other DMD patients. This result was confirmed by comparing ages at loss of ambulation in all non-ambulant DMD patients of the DMD cohort identified in a molecular diagnostic lab. Conclusions: These prospective and retrospective data demonstrate that DMD-53 patients have clinically more severe phenotypes than other DMD patients.

Published
2015

15. Sequential treatment with 5-aza-2′deoxycitidine and deacetylase inhibitors reactivates HIV

Author

Van Lint, C., primary, Bouchat, S., additional, Delacourt, N., additional, Kula, A., additional, Darcis, G., additional, Corazza, F., additional, Gatot, J.S., additional, Melard, A., additional, Vanhulle, C., additional, Van Driessche, B., additional, Kabeya, K., additional, Pardons, M., additional, Avettand-Fenoel, V., additional, Clumeck, N., additional, De Wit, S., additional, Rohr, O., additional, and Rouzioux, C., additional

Published
2015
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23. Pilocytic astrocytoma of the optic pathway: a tumour deriving from radial glia cells with a specific gene signature

Author

Tchoghandjian, A., primary, Fernandez, C., additional, Colin, C., additional, El Ayachi, I., additional, Voutsinos-Porche, B., additional, Fina, F., additional, Scavarda, D., additional, Piercecchi-Marti, M.-D., additional, Intagliata, D., additional, Ouafik, L., additional, Fraslon-Vanhulle, C., additional, and Figarella-Branger, D., additional

Published
2009
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34. THERAPEUTIC AND NUTRITIONAL EFFECT OF AN ORAL REHYDRATION SOLUTION (ORS) CONTAINING GLUTAMINE. A DOUBLE BLIND RANDOMISED STUDY IN 44 DIARRHEIC INFANTS.

Author

Mouterde, O., primary, Mallet, E., additional, Vanhulle, C., additional, Chauvière, G., additional, Hévin, B., additional, Meunier, M., additional, Lenaerts, C., additional, Ginies, J. L., additional, Claeyssens, S., additional, Heckettsweiler, B., additional, and Basuyau, J. P., additional

Published
1997
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42. Syngeneic Adoptive Transfer of Anti-Human Immunodeficiency Virus-1 (HIV-l)-Primed Lymphocytes From a Vaccinated HIV-Seronegative Individual to His HIV-1-Infected Identical Twin

Author

Bex, F., Hermans, P., Sprecher, S., Achour, A., Badjou, R., Desgranges, C., Cogniaux, J., Franchioli, P., Vanhulle, C., Lachgar, A., Stryckmans, P., Zagury, D., Burny, A., and Clumeck, N.

Abstract

Immunotherapy by adoptive transfer of lymphocytes was attempted in identical twins, one who was virus-free and the other who was infected with human immunodeficiency virus-1 (HIV-1), at the stage of acquired immunodeficiency syndrome. The noninfected twin was vaccinated by priming with a recombinant vaccinia virus expressing the envelope glycoprotein of one of his brother’s viruses and boosting with the same purified gp160 adsorbed on alum. Vaccination elicited major histocompatibility complex class I-restricted CD8+cytolytic T lymphocytes specific for HIV-1, but no antibody response. The diseased brother, a 38-year-old homosexual who had developed repeated opportunistic infections since 1990 and had a CD4+count reduced to practically zero, was treated by infusions of lymphocytes collected from the vaccinated brother by lymphopheresis. After a first transfer of the whole lymphocyte population, no changes were observed in the clinical status and biologic or virologic parameters. A second transfer was then applied with activation of the cells with purified envelope glycoprotein before infusion. The outcome of the treatment was an increase in total lymphocytes, in CD4+and activated CD8+DR+cell counts, and in proliferative responses to HIV antigens. A marked but transient 3-log increase in cellular and plasmatic virus loads was also observed after the second adoptive transfer. These observations will be considered with attention to improve the future adoptive transfer protocols, especially in patients with severe CD4+depletion.

Published
1994
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43. Epilepsies and time to diagnosis

Author

Cachera, C., Baulac, M., Fagnani, F., Jallon, P., Leveau, J., Loiseau, P., Motte, J., Thomas, P., Vallee, L., Allaire, C., Autret, A., Baldy-Moulinier, M., Clanet, M., Dordain, G., Gastaut, J. L., Giroud, M., Josien, E., Marescaux, C., Masnou, P., Mauguiere, F., Parain, D., Perret, J., Revol, M., Rumbach, L., Tapie, P., Weber, M., Adam, C., Attal, N., Attane, F., Aubrun, P., Ayrivie, N., Badinand-Hubert, N., Bapst-Reiter, J., Barthez-Carpentier, M. A., Bartolomei, F., Bataillard, M., Bednarek, N., Belair, C., Benazet, M., Berges, S., Bergouignan, F. X., Bernard, C., Bernard-Bourzeix, L., Bertran, F., Bertrand, P., Beuriat, P., Billard, C., Bille-Turc, F., Billy, C., Biraben, A., Blanc, A., Boidein, F., Bouayed, N., Boudon, S., Bouillat, J., Boulloche, J., Bredin, A., Brocard, O., Brosset, P., Brunet-Bourgin, F., Cenraud, B., Chaigne, D., Chaix, Y., Chaunu, M. P., Chavot, D., Clavelou, P., Cohadon, S., Collombier, N., Contis, P. E., Convers, P., Couchot, J., Cournelle, M. A., Courtois, S., Croguennec, J. M., Cuisset, J. M., Cuvellier, J. C., D Anglejan, J., Damon, G., Danielli, A., Daubney, P., Bellescize, J., Lumley, L., Recondo, A., Swarte, M., Deffond, D., Delangre, T., Delisse, B., Derambure, P., Derambure, S., Desbordes, P., Desfrancois, F., Destee-Warot, M., Dien, J., Doremus, B., Dourneau-Lethiecq, M. C., Dubois, F., Duche, B., Ducrocq, X., Duhurt, J., Duprey, J., Durand, G., Dusser, A., Escaillas, J. P., Fanjaud, G., Felten, D., Fischer, C., Fontan, D., Formosa, F., Foulon, E., Furby, A., Gallet, S., Galmiche, J., Garde-Arthaud, P., Garrel, S., Gaultier, C., Gauthier, C., Gauthier-Morel, D., Genton, P., Geraud, G., Girard, J. P., Girard-Madoux, M., Gonnaud, P., Goulon-Goeau, C., Gros, S., Grosclaude, M., Gross, M., Gueguen, B., Guinot, H., Haenggeli, C. A., Hamon, J. B., Henlin, J. L., Hevin, B., Hinault, P., Homeyer, P., Hommet, C., Huart, E., Huc, P., Huttin, B., Inglesiakis, L., Isnard, J., Isnard, H., Jogeix, M., Juhel, C., Kahane, P., Kalafat, M., Keo-Kosal, P., Kreib, A. M., Kubler, C., Larrieu, E., Larrieu, J. L., Latinville, D., Le Gallou-Wittenberg, A., Lebas, F., Lebrun-Grandie, P., Leche, J., Legout, A., Legrand, S., Legroux, M., Lemaitre, J. F., Lestavel, P., Levasseur, M., Lienhard, C., Livet, M. O., Louiset, P., Lubeau, M., Lucas, B., Lucas-Daviaud, J., Maillard, S., Maillet-Vioud, M., Mancini, J., Mann, M., Marchal, C., Martini, L., Maupetit, J., Maynard, R., Menage, P., Menard, D., Metreau, R., Milor, M., Minot-Myhie, M. C., Moene, Y., Montagne, B., Montelescaut, M. E., Moreaud, O., Noelle, B., Olmi, X., Orbegozo, J., Ouvrard-Hernandez, A. M., Parsa, A., Pautrizel, B., Pedespan, J. M., Pernes, P., Perrouty, B., Petit, J., Peudenier, S., Picard, A. M., Pierrot-Deseilligny, C., Planque, E., Portha, C., Preux, P. M., Prud Homme, M., Raybaut-Guilhem, D., Rebaud, P., Regi, A., Regi, J. L., Reis, J., Rejou, F., Remy, C., Renard, J. F., Revenu, M., Revol, A., Rey, M., Richelme, C., Ricou, P., Rigal, J. P., Rogez, R., Rousselle, C., Rummens, C., Philippe Ryvlin, Sabouraud, P., Saikali, I., Saudeau, D., Savet, J. F., Schaeffer, J. L., Schaff, J. L., Schoenfelder, F., Schuermans, P., Senant, J., Setiey, A., Sevrin, C., Sivelle, G., Soisson, T., Soubielle, P., Soulages, X., Soulayrol, S., Tabaraud, F., Taillandier, P., Tannier, C., Tarel, V., Taussig, D., Thedrez, F., Tournier, C. L., Turc, J. D., Vanhulle, C., Vaunaize, J., Verier, A., Vernay, D., Visy, J. M., Vongsouthi, C., Vrigneaud, J., Waubant, E., Weichlein, A., Weill, O., Zai, L., Ziegler, F., Zix, C., Zelicourt, M., and Grp, Carole

44. The DM-scope registry: a rare disease innovative framework bridging the gap between research and medical care

Author

Antonio, M., Dogan, C., Eymard, B., Puymirat, J., Mathieu, J., Gagnon, C., Attarian, S., Ac Aube-Nathier, Audic, F., Bach, N., Barnerias, C., Al Bedat-Millet, Behin, A., Bellance, R., Rabah BEN YAOU, Bombard, V., Bouhour, F., Boutte, C., Boyer, F., Cances, C., Chabrol, B., Jb Chanson, Chapon, F., Chasseriau, R., Cintas, P., Am Cobo, Colombert, V., Mc Cruz, Jm Cuisset, Deschamps, R., Desguerre, I., Durigneux, J., Duval, F., Espil, C., Fafin, C., Feasson, L., Fradin, M., Furby, A., Goldenberg, A., Grotto, S., Ghorab, K., Guyant-Marechal, L., Heron, D., Isapof, A., Jacquin-Piques, A., Journel, H., Laforet, P., Lagrue, E., Laroche-Raynaud, C., Laugel, V., Lebeau, F., Magot, A., Manel, V., Mayer, M., Mercier, S., Menard, D., Michaud, M., Mc Minot, Rj Morales, Nadaj-Pakleza, A., Jb Noury, Pasquier, L., Pellieux, S., Pereon, Y., Perrier, J., Peudenier, S., Preudhomme, M., Pouget, J., Quijano-Roy, S., Ragot-Mandry, S., Richelme, C., Rivier, F., Sabouraud, P., Sacconi, S., Salort-Campana, E., Sarret, C., Schaeffer, S., Sole, G., Stojkovic, T., Taithe, F., Testard, H., Tiffereau, V., Urtizberea, A., Vanhulle, C., Vial, C., Walther-Louvier, U., Zagnoli, F., Hamroun, D., Bassez, G., CHU Pitié-Salpêtrière [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Institut de Myologie, Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Association française contre les myopathies (AFM-Téléthon)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS), Institut de biologie et chimie des protéines [Lyon] (IBCP), Université Claude Bernard Lyon 1 (UCBL), Université de Lyon-Université de Lyon-Centre National de la Recherche Scientifique (CNRS), Centre de Recherches du Service de Santé des Armées (CRSSA), Service de Santé des Armées, Energy Storage and Conversion, Research Institute of Hydro-Québec, Energy Storage and Conversion, Hôpital de la Timone [CHU - APHM] (TIMONE), Department of Medicine, Icahn School of Medicine at Mount Sinai [New York] (MSSM), INVENTAIRE FORESTIER NATIONAL CAEN, Partenaires IRSTEA, Institut national de recherche en sciences et technologies pour l'environnement et l'agriculture (IRSTEA)-Institut national de recherche en sciences et technologies pour l'environnement et l'agriculture (IRSTEA), Centre de référence Caribéen pour les maladies neuromusculaires (CeRCa), Hôpital Pierre Zobda-Quitman [CHU de la Martinique], CHU de la Martinique [Fort de France]-CHU de la Martinique [Fort de France], Centre de recherche en Myologie – U974 SU-INSERM, Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU), Hospices Civils de Lyon (HCL), Contrôle de la Réponse Immune B et des Lymphoproliférations (CRIBL), Université de Limoges (UNILIM)-Génomique, Environnement, Immunité, Santé, Thérapeutique (GEIST FR CNRS 3503)-Centre National de la Recherche Scientifique (CNRS), Centre Hospitalier Universitaire de Toulouse (CHU Toulouse), Service de Neurologie Pédiatrique, Assistance Publique - Hôpitaux de Marseille (APHM)- Hôpital de la Timone [CHU - APHM] (TIMONE), Service de Neurologie [CHU Caen], Université de Caen Normandie (UNICAEN), Normandie Université (NU)-Normandie Université (NU)-CHU Caen, Normandie Université (NU)-Tumorothèque de Caen Basse-Normandie (TCBN)-Tumorothèque de Caen Basse-Normandie (TCBN), Centre de compétences pathologies neuromusculaires [CHU Caen], Département Neurologie [CHU Toulouse], Pôle Neurosciences [CHU Toulouse], Centre Hospitalier Universitaire de Toulouse (CHU Toulouse)-Centre Hospitalier Universitaire de Toulouse (CHU Toulouse), Université Paris Descartes - Paris 5 (UPD5), Université d'Angers (UA), Institut de Recherche en Systèmes Electroniques Embarqués (IRSEEM), Université de Rouen Normandie (UNIROUEN), and Normandie Université (NU)-Normandie Université (NU)-École Supérieure d’Ingénieurs en Génie Électrique (ESIGELEC)

Subjects
[SDV]Life Sciences [q-bio]
Abstract

International audience; Background: The relevance of registries as a key component for developing clinical research for rare diseases (RD) and improving patient care has been acknowledged by most stakeholders. As recent studies pointed to several limitations of RD registries our challenge was (1) to improve standardization and data comparability; (2) to facilitate interoperability between existing RD registries; (3) to limit the amount of incomplete data; (4) to improve data quality. This report describes the innovative concept of the DM-Scope Registry that was developed to achieve these objectives for Myotonic Dystrophy (DM), a prototypical example of highly heterogeneous RD. By the setting up of an integrated platform attractive for practitioners use, we aimed to promote DM epidemiology, clinical research and patients care management simultaneously.Results: The DM-Scope Registry is a result of the collaboration within the French excellence network established by the National plan for RDs. Inclusion criteria is all genetically confirmed DM individuals, independently of disease age of onset. The dataset includes social-demographic data, clinical features, genotype, and biomaterial data, and is adjustable for clinical trial data collection. To date, the registry has a nationwide coverage, composed of 55 neuromuscular centres, encompassing the whole disease clinical and genetic spectrum. This widely used platform gathers almost 3000 DM patients (DM1 n = 2828, DM2 n = 142), both children (n = 322) and adults (n = 2648), which accounts for > 20% of overall registered DM patients internationally. The registry supported 10 research studies of various type i.e. observational, basic science studies and patient recruitment for clinical trials.Conclusion: The DM-Scope registry represents the largest collection of standardized data for the DM population. Our concept improved collaboration among health care professionals by providing annual follow-up of quality longitudinal data collection. The combination of clinical features and biomolecular materials provides a comprehensive view of the disease in a given population. DM-Scope registry proves to be a powerful device for promoting both research and medical care that is suitable to other countries. In the context of emerging therapies, such integrated platform contributes to the standardisation of international DM research and for the design of multicentre clinical trials. Finally, this valuable model is applicable to other RDs.

45. Syngeneic adoptive transfer of anti-human immunodeficiency virus-1 (HIV-1)-primed lymphocytes from a vaccinated HIV-seronegative individual to his HIV-1 infected identical twin

Author

Bex, F., Hermans, P., Sprecher, S., Achour, A., Badjou, R., Desgranges, C., Cogniaux, J., Franchioli, P., Vanhulle, C., Lachgar, A., Stryckmans, P., Zagury, D., Burny, A., and Clumeck, N.

Subjects
HIV infection -- Care and treatment, Immunotherapy -- Usage, AIDS vaccines -- Usage
Abstract

According to the authors' abstract of an article published in Blood, "Immunotherapy by adoptive transfer of lymphocytes was attempted in identical twins, one who was virus-free and the other who [...]

Published
1994

46. DNA cytosine methylation in the Bovine Leukemia Virus promoter is associated with latency in a Lymphoma-derived B-cell line : potential involvement of direct inhibition of CREB/CREM/ATF binding

Author

Van Lint Carine, Rohr Olivier, Hirsch Ivan, Burny Arsène, Portetelle Daniel, Kettmann Richard, Twizere Jean-Claude, Nuttinck Michèle, Merimi Makram, Cardona Christelle, Blazkova Jana, Vanhulle Caroline, Guiguen Allan, Colin Laurence, and Van Driessche Benoit

Subjects
Immunologic diseases. Allergy, RC581-607
Published
2011
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50. Evaluation of professional practices in the use of mexiletine for the management of childhood myotonia in French pediatric neuromuscular centers (MEXI-PEDI survey).

Author

Barrière S, Manel V, Barnerias C, Wahbi K, Audic F, Cances C, Chouchane M, Dabaj I, Davion JB, Desguerre I, Durigneux J, Espil-Taris C, Gousse G, Gitiaux C, Lambert C, Laroche C, Laugel V, Moing AL, Pereon Y, Quijano-Roy S, Ropars J, Sarrazin E, Serrand B, Thibaud M, Trommsdorff V, Urtizberea JA, Vanhulle C, Walther-Louvier U, Isapof A, and Sarret C

Subjects
France, Voltage-Gated Sodium Channel Blockers therapeutic use, Surveys and Questionnaires, Humans, Infant, Child, Preschool, Child, Adolescent, Mexiletine adverse effects, Mexiletine therapeutic use, Pediatrics statistics & numerical data, Myotonia drug therapy, Off-Label Use statistics & numerical data, Neurologists statistics & numerical data, Practice Patterns, Physicians' statistics & numerical data
Abstract

Background: Myotonia is the main feature of both myotonic dystrophy (DM) and non-dystrophic myotonia (NDM). It is felt as stiffness, pain, fatigue, and weakness. In France, mexiletine, a non-selective voltage-gated sodium channel blocker, is approved for the treatment of myotonia in adults with NDM, and it has a temporary recommendation for use in the symptomatic treatment of DM in adults. However, it is not currently licensed for treating myotonia in children due to the lack of studies on its use in pediatrics. This has meant heterogeneous practices in its utilization and has led to prescriber reluctance, which has jeopardized accessibility. We undertook a professional practice survey of French pediatric neuromuscular centers to determine their prescribing habits for mexiletine, assessing indications, doses, efficacy, and tolerance., Methods: One medical pediatric professional from each French pediatric neuromuscular center belonging to the national neuromuscular network (FILNEMUS) was invited to complete an anonymous questionnaire., Results: In total, 34 healthcare professionals responded. Of these, 16 had already treated a child for myotonia with mexiletine. Mexiletine was prescribed in one third of pediatric patients with NDM, but it was used in only 3% of DM type 1 patients and in no DM type 2 patients. Pre-treatment assessment always included a cardiac evaluation; however, the method of introduction (inpatient vs. outpatient basis), dosage adjustment, and efficacy evaluation ranged widely. More than half of the respondents reported a high efficacy of mexiletine in their patients; only three reported moderate adverse events (dyspepsia, loss of appetite, and asthenia)., Conclusion: The findings of this first survey on mexiletine for pediatric myotonia in France lend support for the creation of future national guidelines., Competing Interests: Declaration of competing interest - Christine Barnerias and Arnaud Isapof are investigators in the following clinical study: ClinicalTrials NCT04624750 - Christine Barnerias, Catherine Sarret, Yann Pereon, Karim Wahbi, and Arnaud Isapof have participated in industrial symposia for, or have been on the scientific board of, Lupin Pharmaceuticals., (Copyright © 2025. Published by Elsevier Masson SAS.)

Published
2025
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