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Chemical Structure| 775304-57-9 Chemical Structure| 775304-57-9

Structure of Ataluren
CAS No.: 775304-57-9

Chemical Structure| 775304-57-9

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PTC-124 is nonsense allele inhibitor, used to treat Duchenne muscular dystrophy.

Synonyms: PTC124

4.5 *For Research Use Only !

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Product Details of Ataluren

CAS No. :775304-57-9
Formula : C15H9FN2O3
M.W : 284.24
SMILES Code : O=C(O)C1=CC=CC(C2=NOC(C3=CC=CC=C3F)=N2)=C1
Synonyms :
PTC124
MDL No. :MFCD09864996
InChI Key :OOUGLTULBSNHNF-UHFFFAOYSA-N
Pubchem ID :11219835

Safety of Ataluren

GHS Pictogram:
Signal Word:Warning
Hazard Statements:H302-H315-H319-H332-H335
Precautionary Statements:P261-P280-P305+P351+P338

Isoform Comparison

Biological Activity

Target
  • CFTR

Clinical Trial:

NCT Number Conditions Phases Recruitment Completion Date Locations
NCT03256799 Cystic Fibrosis Phase 4 Completed - United States, Alabama ... More >> University of Alabama at Birmingham Birmingham, Alabama, United States, 35233 Less <<
NCT02139306 Cystic Fibrosis Phase 3 Unknown November 2016 -
NCT01182324 - Completed - United States, Maryland ... More >> National Human Genome Research Institute (NHGRI), 9000 Rockville Pike Bethesda, Maryland, United States, 20892 Less <<
NCT02369731 - Recruiting April 2022 Austria ... More >> Krankenhaus der Barmherzigen Schwestern Linz Recruiting Linz, Austria, 4010 Principal Investigator: Veronika Pilshofer, Dr          Preyer Children Hospital Recruiting Wien, Austria, 1100 Principal Investigator: Guenther Bernert, Pr          France CHU Brest, hôpital Morvan Recruiting Best, France, 29609 Principal Investigator: Ropars Juliette, Dr          CHU Bordeaux Pelgrin Recruiting Bordeaux Cedex, France, 33076 Principal Investigator: Caroline Espil-Taris          L'Escale Hôpital Femme Mère Enfant- Hospices Civils de Lyon Recruiting Bron, France, 69500 Principal Investigator: Carole Vuillerot, Dr          CHU Clermont-Ferrand, hôpital Estaing Recruiting Clermont-Ferrand, France, 63003 Principal Investigator: Catherine Sarret, Dr          CHU de Martinique - Hôpital P. Zobda-Quitman Recruiting Fort De France Cedex, France, 97261 Principal Investigator: Rémi Bellance, Dr          CHRU de Lille Recruiting Lille Cedex, France, 59037 Principal Investigator: Jean-Marie Cuisset, Dr          CHU Marseille Hôpital de la Timone Recruiting Marseille Cedex 5, France, 13385 Principal Investigator: Brigitte Chabrol, Pr          CHU Nantes Recruiting Nantes, France, 44000 Principal Investigator: Yan Pereon, Pr          Hôpital Armand Trousseau Recruiting Paris, France, 75012 Principal Investigator: Gidaro Teresa, Dr          Hôpital Necker Recruiting Paris, France, 75015 Principal Investigator: Isabelle Desguerre, Pr          CHU Reims Recruiting Reims, France, 51100 Principal Investigator: Pascal Sabouraud, Dr          CHU Saint-Etienne MPR pédiatrique Hôpital Bellevue Recruiting Saint Etienne Cedex, France, 42055 Principal Investigator: Stéphane Chabrier, Dr          CHU de la Réunion - GHSR - GH Sud-Réunion Recruiting Saint Pierre, France, 97448 Principal Investigator: Renouil Michel, Dr          CHU Strasbourg Recruiting Strasbourg Cedex, France, 67098 Principal Investigator: Vincent Laugel, Pr          CHU Toulouse Recruiting Toulouse, France, 31059 Principal Investigator: Pascal Cintas, Dr          Hôpital d'enfants Gatien de Clocheville Recruiting Tours Cedex 9, France, 37044 Principal Investigator: Lagrue Emmanuelle, Dr          CHU Nancy Hôpital de Brabois-Enfants Rue du Morvan Recruiting Vandoeuvre-les-Nancy, France, 54500 Principal Investigator: Emmanuel Raffo, Pr          Germany Klinikum Bayreuth GmbH Recruiting Bayreuth, Germany, 95445 Principal Investigator: Almut Hirsch, Dr          Charité Universitätsmedizin Berlin Recruiting Berlin, Germany, 13353 Principal Investigator: Claudia Weiss, Dr          Universitätsklinikum Erlangen Recruiting Erlangen, Germany, 91054 Principal Investigator: Regina Trollmann, Pr          Universitaetsklinikum Essen (AoR) Recruiting Essen, Germany, 45147 Principal Investigator: Ulrike Schara, Pr          SPZ Frankfurt Mitte Recruiting Frankfurt am Main, Germany, 60316 Principal Investigator: Jurgen Seeger, Dr          Universitaetsklinikum Freiburg Recruiting Freiburg, Germany, 79106 Principal Investigator: Janbernd Kirschner, Pr          UKE, Universitätsklinikum Hamburg-Eppendorf Recruiting Hamburg, Germany, 20246 Principal Investigator: Jessica Johannsen, Dr          Israel Hadassah Medical Center Recruiting Jerusalem, Israel, 91120 Principal Investigator: Talya Dor, Dr          Chaim Sheba Medical Center Ramat Gan, Neuromuscle clinic Recruiting Ramat Gan, Israel, 52621 Principal Investigator: Amir Dori, Dr          Sweden Queen Silvia Children's Hospital Recruiting Goteborg, Sweden, 41685 Principal Investigator: Mar Tulinius, Pr Less <<
NCT00237380 Cystic Fibrosis PHASE2 COMPLETED 2006-05-31 Hadassah University Hospital -... More >> Mount Scopus, Jerusalem, 91240, Israel Less <<
NCT00458341 Cystic Fibrosis PHASE2 COMPLETED 2008-02-29 Reine Fabiola Hospital, Brusse... More >>ls, 1020, Belgium|UZ Gasthuisberg Leuven, Leuven, 3000, Belgium|Hopital Necker Enfants Malades, Paris, 75015, France Less <<
NCT00264888 Duchenne Muscular Dystrophy PHASE2 COMPLETED 2025-05-07 Cincinnati Children's Hospital... More >> Medical Center, Cincinnati, Ohio, 45229-3039, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104-4399, United States|University of Utah, Salt Lake City, Utah, 84112, United States Less <<
NCT03256968 Cystic Fibrosis PHASE4 COMPLETED 2018-12-31 University of Alabama at Birmi... More >>ngham, Birmingham, Alabama, 35233, United States Less <<
NCT00234663 Cystic Fibrosis PHASE2 COMPLETED 2025-08-06 University of Alabama at Birmi... More >>ngham, Birmingham, Alabama, 35233-1711, United States|Stanford University Medical Center, Palo Alto, California, 94304-5786, United States|The Children's Hospital, Denver, Colorado, 80218, United States|Johns Hopkins Hospital, Baltimore, Maryland, 21287, United States|Rainbow Babies and Children's Hospital, Cleveland, Ohio, 44106, United States Less <<
NCT03179631 Muscular Dystrophy, Duchenne ... More >> Muscular Dystrophies Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Disease Neuromuscular Diseases Nervous System Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Less << Phase 3 Recruiting December 31, 2021 -
NCT02409004 Healthy Phase 1 Completed - Canada ... More >> Inventiv Quebec, Canada, G1P 0A2 Less <<
NCT00351078 Cystic Fibrosis PHASE2 COMPLETED 2025-07-07 Hadassah University Hospital -... More >> Mount Scopus, Jerusalem, 91240, Israel Less <<
NCT02647359 Aniridia Phase 2 Active, not recruiting December 2019 United States, Oregon ... More >> Casey Eye Institute, Oregon Health & Science University Portland, Oregon, United States, 97239 United States, Virginia University of Virginia Charlottesville, Virginia, United States, 22908 Canada, British Columbia University of British Columbia Vancouver, British Columbia, Canada, V5Z3N9 Less <<
NCT00592553 Duchenne Muscular Dystrophy ... More >> Becker Muscular Dystrophy Less << Phase 2 Completed - -
NCT01141075 Amino Acid Metabolism, Inborn ... More >>Errors Less << PHASE2 TERMINATED 2011-11-03 ZNA Queen Paola Child Hospital... More >> and Provincial Centre for Metabolic Disorders, Antwerp, Belgium|H?pital Edouard Herriot, Lyon, France|Necker-Enfants Malades Hospital, Paris, France|University Children's Hospital, Duesseldorf, Germany|Istituti Clinici di Perfezionamento, Milano, Milan, Italy|Federico II University, Naples, Italy|University Hospital, Department of Pediatrics, Padova, Italy|University Children's Hospital, Zürich, Switzerland|Great Ormand Street Hospital, London, United Kingdom Less <<
NCT00947193 Hemophilia A|Hemophilia B PHASE2 TERMINATED 2011-08-30 The Bleeding and Clotting Diso... More >>rders Institute, Peoria, Illinois, 61614, United States|St. Vincent Indianapolis Hospital, Indianapolis, Indiana, 46260, United States|New England Hemophilia Center, Worcester, Massachusetts, 01605, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Vanderbilt Hemostatis and Thrombosis Clinic, Nashville, Tennessee, 37232, United States|Puget Sound Blood Center, Seattle, Washington, 98104, United States|St. Paul's Hospital, Vancouver, British Columbia, V6Z 1Y6, Canada|H?pital Cardiologique, Lille Cedex, France|H?pital Edouard Herriot, Lyon Cedex, France|H?pital Necker Enfants Malades, Paris, France|Azienda Ospedaliero-Universitaria Careggi Viale G.B. Morgagni, Firenze, Italy|A.Bianchi Bonomi Hemophilia and Thrombosis Center, Milano, Italy Less <<
NCT01140451 Cystic Fibrosis PHASE3 COMPLETED 2013-12-02 University of Alabama-Birmingh... More >>am, Birmingham, Alabama, 35233, United States|Miller Children's Hospital Long Beach, Long Beach, California, 90806, United States|Lucile Packard Children's Hospital, Palo Alto, California, 94304, United States|The Children's Hospital, Aurora, Colorado, 80045, United States|University of Miami, Miami, Florida, 33136, United States|Emory University Cystic Fibrosis Center, Atlanta, Georgia, 30322, United States|Children's Memorial Hospital, Chicago, Illinois, 60614, United States|Johns Hopkins Children's Center, Baltimore, Maryland, 21287, United States|Children's Hospital Boston, Boston, Massachusetts, 02115, United States|Washington University, Saint Louis, Missouri, 63110, United States|St. Vincent's Hospital, New York, New York, 10011, United States|New York Medical College, Valhalla, New York, 10595, United States|University of North Carolina, Chapel Hill, North Carolina, 27599, United States|Rainbow Babies & Children's Hospital, Cleveland, Ohio, 44106, United States|Childrens Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15213, United States|H?pital Erasme, Brussels, Belgium|H?pital Universitaire des Enfants Reine Fabiola, Brussels, Belgium|University Hospital Brussels, Brussels, Belgium|University Hospital Leuven, Leuven, Belgium|University of Toronto, Toronto, Canada|H?pital Cochin, Paris, France|H?pital Necker - Enfants Malades, Paris, France|H?pital des Enfants, Toulouse, 31059, France|Klinikum der Universit?t K?ln, K?ln, Germany|Hadassah University Hospital - Mount Scopus, Jerusalem, 91240, Israel|Università La Sapienza, Roma, Italy|Azienda Ospedaliera di Verona, Verona, Italy|Universitair Medisch Centrum Utrecht, Utrecht, Netherlands|Hospital Universitario La Paz, Madrid, Spain|Karolinska University Hospital, Huddinge, Stockholm, Sweden|Belfast City Hospital, Belfast, United Kingdom Less <<
NCT01826487 Muscular Dystrophy, Duchenne ... More >> Muscular Dystrophies Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Less << Phase 3 Completed - -
NCT00759876 Duchenne Muscular Dystrophy PHASE2 TERMINATED 2010-05-17 Cincinnati Children's Hospital... More >> Medical Center, Cincinnati, Ohio, 45229-3039, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|University of Utah, Salt Lake City, Utah, 84112, United States Less <<
NCT00803205 Cystic Fibrosis Phase 3 Completed - -
NCT02758626 Epilepsy Phase 2 Active, not recruiting December 2022 United States, New York ... More >> New York University School of Medicine New York, New York, United States, 10016 Less <<
NCT01247207 Duchenne Muscular Dystrophy PHASE3 ENROLLING_BY_INVITATION 2025-04-30 Phoenix Childrens Hospital, Ph... More >>oenix, Arizona, 85016, United States|Children's Hospital of Los Angeles, Los Angeles, California, 90027, United States|UCLA, Los Angeles, California, 90095, United States|Stanford University, Palo Alto, California, 94304, United States|University of California-Davis, Sacramento, California, 95817, United States|University of CA, San Francisco-Benioff Children's Hospital, San Francisco, California, 94158, United States|Children's Hospital Colorado - Center for Cancer and Blood Disorders, Aurora, Colorado, 80045, United States|Child Neurology Center of NW Florida NW, Gulf Breeze, Florida, 32561, United States|Rare Disease Research, LLC, Atlanta, Georgia, 30329, United States|Rush Univ Medical Center, Chicago, Illinois, 60612, United States|University of Iowa Children's Hospital, Iowa City, Iowa, 52242, United States|University of Kansas Medical Center, Kansas City, Kansas, 66160, United States|Children's Hospital of Boston/Harvard Medical School, Boston, Massachusetts, 02115, United States|University of Michigan, Ann Arbor, Michigan, 48109, United States|University of Minnesota, Minneapolis, Minnesota, 55455, United States|Washington University Medical School, Saint Louis, Missouri, 63110, United States|Columbia University Pediatric Neuromuscular Center, New York, New York, 10032, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Cincinnati Childrens Hospital Medical Center, Cincinnati, Ohio, 45229, United States|Nationwide Children's Hospital, Columbus, Ohio, 43205, United States|Shriners Hospital for Children-Portland, Portland, Oregon, 97239, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, 16148, United States|Childrens Medical Center Dallas, Dallas, Texas, 75207, United States|Texas Children's Hospital, Houston, Texas, 77030, United States|University of Texas Health Science Center at San Antonio, San Antonio, Texas, 78229, United States|University of Utah, Salt Lake City, Utah, 84112, United States|Children's Hospital of the King's Daughters, Norfolk, Virginia, 23507, United States|Seattle Children's Hospital, Seattle, Washington, 98105, United States|Alberta Children's Hospital, Calgary, Alberta, Canada|British Columbia Children's Hospital, Vancouver, British Columbia, Canada|London Health Sciences Centre, London, Ontario, Canada|Children's Hospital of East Ontario, Ottawa, Ontario, Canada|CHU de Québec - Université Laval, Quebec, Canada Less <<
NCT03648827 Duchenne Muscular Dystrophy PHASE2 COMPLETED 2020-10-23 Phoenix Childrens Hospital, Ph... More >>oenix, Arizona, 85016, United States|University of California, Los Angeles (UCLA), Los Angeles, California, 90025, United States|University of California (UC) Davis Medical Center, Sacramento, California, 95817, United States|University of Kansas Medical Center, Kansas City, Kansas, 66160, United States|University of Minnesota, Minneapolis, Minnesota, 55455, United States|Columbia University College of Physicians & Surgeons, New York, New York, 10032, United States|Texas Children's Hospital, Houston, Texas, 77030, United States|University of Texas Heath Science Center at San Antonio, San Antonio, Texas, 78229-3900, United States|Children's Hospital of the King's Daughters, Norfolk, Virginia, 23507, United States Less <<
NCT00847379 Duchenne Muscular Dystrophy|Be... More >>cker Muscular Dystrophy Less << PHASE2 TERMINATED 2010-05-24 University of California - Dav... More >>is, Sacramento, California, 95817, United States|Department of Rehabilitation, The Children's Hospital, Aurora, Colorado, 80045, United States|Child Neurology Center of NW Florida, Gulf Breeze, Florida, 32561, United States|University of Iowa Children's Hospital, Division of Child Neurology, Iowa City, Iowa, 52242, United States|University of Kansas Medical Center, Kansas City, Kansas, 66160, United States|Children's Hospital of Boston, Boston, Massachusetts, 02115, United States|University of Minnesota, Minneapolis, Minnesota, 55414, United States|Washington University Medical School, Saint Louis, Missouri, 63110, United States|Columbia University, New York, New York, 10032, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States|Shriners Hospital for Children-Portland, Portland, Oregon, 97239, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Southwestern University, Dallas, Texas, 75207, United States|University of Utah, Salt Lake City, Utah, 84112, United States|The Royal Children's Hospital, Parkville, Victoria, 3052, Australia|The Children's Hospital at Westmead, Westmead, 2145, Australia|UZ Leuven, Leuven, Belgium|Alberta Children's Hospital, London, Ontario, Canada|Children's Hospital of Western Ontario, London, Ontario, Canada|British Colombia Children's Hopsital, Vancouver, Canada|Hopital d'Enfants CHU Timone, Marseille cedex 20, 13385, France|Laboratoire d'Exploration Fonctionnelles, Nantes cedex 1, France|Groupe Hospitalier Pitie-Salpetriere, Institut de Myologie, Paris, France|University of Essen - Clinic for Children, Essen, Germany|University Hospital, Freiburg, Germany|Hadassah University Hopspital, Jerusalem, Israel|Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena, Milano, 20122, Italy|Ospedale Pediatrico Bambino Gesu, Roma, 00165, Italy|U.O. Complessa di Neuropsichiatria Infantile-Policlinico A. Gemelli-Universita Cattolica, Roma, 00168, Italy|Hospital Sant Joan de Deu, Barcelona, Spain|Hospital Universitario La Fe, Valencia, Spain|Queen Silvia Children's Hospital, Goteborg, S-416 85, Sweden|Astrid Lindgren Pediatric Hospital, Stockholm, Sweden|UCL Instititute of Child Health, Dubowitz, London, United Kingdom|University of Newcastle, Newcastle Upon Tyne, United Kingdom|Robert Jones & Agnes Hunt Orthopaedic Hospital, Oswestry, United Kingdom Less <<
NCT02456103 Cystic Fibrosis PHASE3 TERMINATED 2017-06-02 University of Alabama at Birmi... More >>ngham, Birmingham, Alabama, 35233, United States|Miller Children's Hospital Long Beach, Long Beach, California, 90806, United States|Children's Hospital Los Angeles, Los Angeles, California, 90027, United States|Children's Hospital and Research Center at Oakland, Oakland, California, 94609, United States|Stanford University-Children's Hospital, Palo Alto, California, 94304, United States|Children's Hospital Colorado, Aurora, Colorado, 80045, United States|Nemours Children's Clinic, Jacksonville, Florida, 32207, United States|University of Miami, Miami, Florida, 33136, United States|Miami Children's Hospital, Miami, Florida, 33155, United States|All Children's Hospital, Saint Petersburg, Florida, 33701, United States|Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|Indiana University, Indianapolis, Indiana, 46202, United States|Children's Hospital Boston, Boston, Massachusetts, 02115, United States|Washington University, Saint Louis, Missouri, 63110, United States|Morristown Medical Center, Morristown, New Jersey, 07960, United States|Beth Israel Medical Center, New York, New York, 10011, United States|New York University Langone Medical Center, New York, New York, 10016, United States|Columbia University Medical Center, New York, New York, 10032, United States|University of Cincinnati, Cincinnati, Ohio, 45221, United States|Penn State Milton S. Hershey Medical Center, Hershey, Pennsylvania, 17033, United States|Drexel University College of Medicine, Philadelphia, Pennsylvania, 19129, United States|Texas Children's Hospital, Houston, Texas, 77094, United States|University of Texas Health Science Center, Tyler, Texas, 75708, United States|Childrens Hospital of Wisconsin, Milwaukee, Wisconsin, 53226, United States|Hospital de Ninos Ricardo Gutierrez, Buenos Aires, 1330, Argentina|Hospital Universitario Austral, Buenos Aires, Argentina|Royal Adelaide Hospital, Adelaide, 5000, Australia|Prince Charles Hospital, Chermside, 4032, Australia|Princess Margaret Hospital, Perth, 6840, Australia|University Hospital Brussels, Brussels, 1090, Belgium|Hopital Universitaire des Enfants Reine Fabiola, Brussels, 15, Belgium|University Hospital Leuven, Leuven, 3000, Belgium|Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul, Porto Alegre, Brazil|University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD, Plovdiv, Bulgaria|University Multiprofile Hospital for Active Treatment Aleksandrovska EAD, Sofia, Bulgaria|Clinical Research Institute of Montreal, Montreal, H2W 1R7, Canada|University of Toronto Hospital for Sick Children, Toronto, M5G 1X8, Canada|British Columbia Children's Hospital, Vancouver, V6H 3V4, Canada|Hoptial Arnaud de Villeneuve, Montpellier, 34295, France|Hopital Necker - Enfants Malades, Paris, 75015, France|Centre de Perharidy, Roscoff, 29684, France|Centre Hospitalier Regional Sud Reunion, Saint-Pierre, 97448, France|Charite-Universitatsmedizin Berlin, Berlin, 10117, Germany|St. Josef Hospital GmbH, Bochum, 44791, Germany|University of Cologne Children's Hospital, Cologne, 50937, Germany|Christiane Herzog CF-Zentrum, Frankfurt am Main, 60590, Germany|Universitatsklinikum Jena, Jena, 1, Germany|Dr. Von Haunersches Kinderspital, Munchen, 80337, Germany|LMU Klinikum der Universitat Muchen, Munich, 80539, Germany|Ippokratio General Hospital Of Thessaloniki, Thessaloniki, 541, Greece|Meyer Children's Hospital, Haifa, 31096, Israel|Hadassah University Hospital, Jerusalem, 91240, Israel|Azienda Ospedaliero Universitaria Ospedall Riuniti di Acona-Umberto I G.M. Lancisi G. Salesi, Ancona, 71, Italy|Azienda Ospedaliera A Meyer, Firenze, 50139, Italy|Lombardia Cystic Fibrosis Center, Milan, 20122, Italy|Azienda Policlinico Umberto I, Rome, 00161, Italy|Ospedale Pediatrico Bambino Gesu, Rome, 4, Italy|Azienda Ospedaliera di Verona, Verona, Italy|Hagaziekenhuis, s-Gravenweg, Zuid-Holland, 2545 CH, Netherlands|Radboud University, Nijmegen, 6525 GA, Netherlands|Szpital Dzieciecy Polanki im Macieja Plazynskiego w Gdansku, Gdansk, Poland|Institute of Mother and Child, Warsaw, 01-211, Poland|Hospital Universitario Vall d'Hebron, Barcelona, 08035, Spain|Hospital University, Barcelona, 08035, Spain|Hospital San Juan, Esplugues De Llobregat, 08950, Spain|Hospital Regional Universitario de Malaga, Malaga, 29001, Spain|Hospital de Sabadell, Consorci Sanitari Parc Tauli, Sabadell, 08208, Spain|Hospital Universitario Virgen del Rocio, Sevilla, 41013, Spain|St James University Hospital, Leeds, LS9 7TF, United Kingdom|Royal Brompton Hospital, London, SW3 6NP, United Kingdom Less <<
NCT02819557 Duchenne Muscular Dystrophy PHASE2 COMPLETED 2018-02-09 Child Neuro NWF, Gulf Breeze, ... More >>Florida, 32561, United States|Rush University Medical Center, Chicago, Illinois, 60612, United States|Children's Hospital Boston, Boston, Massachusetts, 02115, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States|Children's Medical Center Dallas, Dallas, Texas, 75390-8843, United States|University of Utah, Salt Lake City, Utah, 84112, United States Less <<
NCT01009294 Duchenne Muscular Dystrophy ... More >> Becker Muscular Dystrophy Less << Phase 2 Terminated - United States, California ... More >> University of California-Davis Davis, California, United States, 95616 United States, Massachusetts Children's Hospital of Boston Boston, Massachusetts, United States, 02115 United States, Minnesota University of Minnesota Minneapolis, Minnesota, United States, 55455 United States, Missouri Washington University Medical School Saint Louis, Missouri, United States, 63110 United States, Ohio Nationwide Children's Hospital Columbus, Ohio, United States, 43205 United Kingdom University of Newcastle Newcastle upon Tyne, United Kingdom, NE1 3BZ Less <<
NCT01557400 Duchenne Muscular Dystrophy|Be... More >>cker Muscular Dystrophy|Dystrophinopathy Less << PHASE3 COMPLETED 2018-01-19 Royal Children's Hospital, Par... More >>kville, Melbourne, Australia|Institute For Neuromuscular Research, The Children's Hospital at Westmead, Westmead, Australia|University Hospital KU Leuven, Leuven, Belgium|Alberta Children's Hospital, Calgary, Alberta, Canada|British Columbia Children's Hospital, Vancouver, British Columbia, Canada|Children's Hospital of Western Ontario, London, Ontario, Canada|H?pital d'Enfants CHU Timone, Marseille, France|Laboratoire d'Exploration Fonctionnelles, Nantes, France|Groupe Hospitalier La Pitie-Salpetriere, Paris, France|University of Essen - Clinic for Children, Essen, Germany|University Hospital, Freiburg, Germany|Hadassah Medical Center, Hebrew University Hospital, Jerusalem, Israel|Ospedale Maggiore Policlinico in Milan, Milan, Italy|Ospedale Pediatrico Bambino Gesu, Rome, Italy|U.O. Complessa di Neuropsichiatria Infantile, Rome, Italy|Hospital Sant Joan de déu, Barcelona, Spain|Hospital Universitari La Fe, Valencia, Spain|Queen Silvia Children's Hospital, G?teborg, Sweden|Astrid Lindgren Pediatric Hospital, Stockholm, Sweden|Great Ormond Street Hospital, London, United Kingdom|University of Newcastle Institute of Human Genetics, Newcastle Upon Tyne, United Kingdom Less <<
NCT02107859 Cystic Fibrosis PHASE3 TERMINATED 2017-06-05 University of Alabama-Birmingh... More >>am, Birmingham, Alabama, 35233, United States|Miller Children's Hospital Long Beach, Long Beach, California, 90806, United States|Denver Children's Hospital, Aurora, Colorado, 80045, United States|Children's Hospital Chicago, Chicago, Illinois, 60614, United States|Children's Hospital Boston, Boston, Massachusetts, 02115, United States|Beth Israel Medical Center, New York, New York, 10003, United States|Rainbow Babies & Children's Hospital, Cleveland, Ohio, 44106, United States|H?pital Universitaire des Enfants Reine Fabiola, Brussels, Belgium|University Hospital Brussels, Brussels, Belgium|University Hospital Leuven, Leuven, Belgium|H?pital Necker - Enfants Malades, Paris, France|H?pital des Enfants, Toulouse, 31059, France|Hadassah University Hospital - Mount Scopus, Jerusalem, 91240, Israel|Università La Sapienza, Roma, Italy|Azienda Ospedaliera di Verona, Verona, Italy|Hospital Universitario La Paz, Madrid, Spain|Karolinska University Hospital, Huddinge, Stockholm, Sweden Less <<
NCT02090959 Muscular Dystrophy, Duchenne|M... More >>uscular Dystrophies|Muscular Disorders, Atrophic|Muscular Diseases|Musculoskeletal Diseases|Neuromuscular Diseases|Nervous System Diseases|Genetic Diseases, X-Linked|Genetic Diseases, Inborn Less << PHASE3 TERMINATED 2018-06-12 University of California, Los ... More >>Angeles, Los Angeles, California, 90095, United States|UC Davis Medical Center, Sacramento, California, 95817, United States|Stanford University Medical Center, Stanford, California, 94305, United States|Children's Hospital Colorado - Center for Cancer and Blood Disorders, Aurora, Colorado, 80045, United States|Child Neurology Center of Northwest Florida, Gulf Breeze, Florida, 32561, United States|Rush University Medical Center, Chicago, Illinois, 60612, United States|University of Iowa, Iowa City, Iowa, 52242, United States|University of Kansas Medical Center, Kansas City, Kansas, 66160, United States|Children's Hospital Boston, Boston, Massachusetts, 02115, United States|University of Minnesota, Minneapolis, Minnesota, 55455, United States|Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research, Saint Louis, Missouri, 63110, United States|Columbia University College of Physicians & Surgeons, New York, New York, 10032, United States|Children's Hospital Cincinnati, Cincinnati, Ohio, 45229, United States|Nationwide Children's Hospital, Columbus, Ohio, 43209, United States|Oregon Health & Science University, Portland, Oregon, 97239, United States|The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15224, United States|Childrens Medical Center Dallas, Texas, Dallas, Texas, 75207, United States|Texas Children's Hospital, Houston, Texas, 77030, United States|University of Utah, Salt Lake City, Utah, 84112, United States|Seattle Children's Hospital - Childhood Cancer and Blood Disorders, Seattle, Washington, 98105, United States|The Children's Hospital at Westmead, Westmead, New South Wales, 2145, Australia|The Royal Children's Hospital, Parkville, Victoria, 3052, Australia|UZ Leuven, Leuven, 3000, Belgium|Universidade Federal do Rio de Janeiro - Instituto de Puericultura e Pediatria Martagao Gesteira, Rio de Janeiro, 21.941-912, Brazil|Sao Paulo University -HC/FMUSP, S?o Paulo, 05403-900, Brazil|Aleksandrovska Hospital, Sofia, 1431, Bulgaria|Alberta Children's Hospital, Calgary, Alberta, T3B 6A8, Canada|British Columbia Children's Hospital, Vancouver, British Columbia, V6H 3V4, Canada|Children's Hospital of Western Ontario, London, Ontario, N6C 2RC, Canada|Hospital Luis Calvo Mackenna, Santiago, Región Metropolitana, Chile|Hospital Clinico Universidad Catolica, Santiago, 8330073, Chile|University Hospital Brno, Brno, 635 00, Czechia|Motol University Hospital, Praha, 150 06, Czechia|Hospital de la Timone, Marseille, 13385, France|CHU de Nantes, Nantes Cedex 1, 44093, France|Groupe Hospitalier Pitie-Salpetriere, Paris Cedex 13, 75651, France|Hopital Necker - Enfants Malades, Paris, 75015, France|Universitaetsklinikum Essen, Essen, 45122, Germany|University Medical Center Freiburg, Freiburg, 79106, Germany|Universitat Munchen - von Haunersche Kinder Clinic, Munchen, 80337, Germany|Hadassah University Hospital, Jerusalem, 91240, Israel|Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano, Milano, 20122, Italy|Bambino Gesu Hospital, Rome, 00165, Italy|U.O. Complessa di Neuropsichiatria Infantile, Rome, 00168, Italy|Policlinico Universitario G. Martino, Sicily, 98125, Italy|Seoul National University Hospital, Seoul, 110-744, Korea, Republic of|Medical University of Warsaw, Warsaw, 502-097, Poland|Hospital Sant Joan de Deu, Barcelona, 08950, Spain|Hospital Universitari i Politecnic la Fe, Valencia, 46026, Spain|Queen Silvia Children's Hospital, Goteburg, SE-416 85, Sweden|Astrid Lindgren Childrens Hospital, Stockholm, 17176, Sweden|CHUV Lausanne, Lausanne, 1011, Switzerland|Hacettepe Childrens Hospital, Ankara, 06100, Turkey|Erciyes University Faculty of Medicine, Talas/Kayseri, 38039, Turkey|University College London Institute of Child Health, London, WC1N 3JH, United Kingdom|Royal Manchester Children's Hospital, Manchester, M13 9WL, United Kingdom|The Newcastle upon Tyne Hospitals, NHS Foundation Trust, Newcastle upon Tyne, NE1 3BZ, United Kingdom Less <<
NCT04336826 Nonsene Mutation Duchenne Musc... More >>ular Dystrophy Less << PHASE2 COMPLETED 2023-08-07 Rare Disease Research, LLC, At... More >>lanta, Georgia, 30329, United States Less <<
NCT04117880 Aniridia PHASE2 WITHDRAWN 2021-01-31 -
NCT03796637 Duchenne Muscular Dystrophy PHASE2 COMPLETED 2019-06-03 University of California, Los ... More >>Angeles (UCLA), Los Angeles, California, 90025, United States Less <<

Protocol

Bio Calculators
Preparing Stock Solutions 1mg 5mg 10mg

1 mM

5 mM

10 mM

3.52mL

0.70mL

0.35mL

17.59mL

3.52mL

1.76mL

35.18mL

7.04mL

3.52mL

References

 

Historical Records

Categories