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Chemical Structure| 1825352-65-5 Chemical Structure| 1825352-65-5

Structure of Risdiplam
CAS No.: 1825352-65-5

Chemical Structure| 1825352-65-5

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Risdiplam is an orally administered, centrally and peripherally distributed SMN2 pre-mRNA splicing modifier that increases survival motor neuron (SMN) protein levels.

Synonyms: RG7916; RO7034067; RO703406

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Product Details of Risdiplam

CAS No. :1825352-65-5
Formula : C22H23N7O
M.W : 401.46
SMILES Code : O=C1C=C(C2=NN3C(C(C)=C2)=NC(C)=C3)N=C4N1C=C(N(C5)CCNC65CC6)C=C4
Synonyms :
RG7916; RO7034067; RO703406
MDL No. :MFCD31657372
InChI Key :ASKZRYGFUPSJPN-UHFFFAOYSA-N
Pubchem ID :118513932

Safety of Risdiplam

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

Related Pathways of Risdiplam

DNA

Isoform Comparison

Biological Activity

Clinical Trial:

NCT Number Conditions Phases Recruitment Completion Date Locations
NCT03040635 Healthy Volunteers Phase 1 Completed - United States, California ... More >> Collaborative Neuroscience Network, Inc. Garden Grove, California, United States, 92845 Less <<
NCT02908685 Muscular Atrophy, Spinal Phase 2 Phase 3 Active, not recruiting July 12, 2020 -
NCT02633709 Spinal Muscular Atrophy Phase 1 Completed - Netherlands ... More >> Pra International Group B.V Groningen, Netherlands, 9728 NZ Less <<
NCT03036501 Healthy Phase 1 Completed - Netherlands ... More >> Pra International Group B.V Groningen, Netherlands, 9728 NZ Less <<
NCT02913482 Muscular Atrophy, Spinal PHASE2 COMPLETED 2023-12-22 Stanford University Medical Ce... More >>nter, Palo Alto, California, 94304, United States|Ann and Robert H. Lurie Children Hospital of Chicago, Chicago, Illinois, 60611, United States|Boston Childrens Hospital, Boston, Massachusetts, 02115, United States|Columbia University Medical Center; The Neurological Institute of New York, New York, New York, 10032, United States|UZ Gent, Gent, 9000, Belgium|UZ Leuven Gasthuisberg, Leuven, 3000, Belgium|Chr de La Citadelle, Liège, 4000, Belgium|Instituto de Puericultura E Pediatria Martag?o Gesteira, Rio de Janeiro, RJ, CEP 21941-912, Brazil|Hospital das Clinicas - FMUSP_X; Neurologia, Sao Paulo, SP, 05403-000, Brazil|Peking University First Hospital, Beijing City, 100034, China|Children's Hospital of Fudan University, Shanghai, 201102, China|Clinical Medical Center Zagreb; University Hospital Rebro Department of Paediatrics, Zagreb, 10000, Croatia|Hopital Femme Mere Enfant; Medecine Physique et Readaptation Pediatrique ? L?ESCALE, Bron, 69677, France|Hopital Armand Trousseau, Paris, 75571, France|IRCCS Ospedale Pediatrico Bambino Gesù; U.O. Malattie Neuromuscolari e Neurodegenerative, Roma, Lazio, 00165, Italy|Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile, Roma, Lazio, 00168, Italy|IRCCS Istituto Giannina Gaslini; U.O.S.D. Centro di Miologia e Patologie Neurodegenerative, Genova, Liguria, 16147, Italy|Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Unità Operativa Complessa di Neurologia, Milano, Lombardia, 20122, Italy|Fondazione IRCCS Istituto Neurologico ?Carlo Besta?; UO di Neurologia dello Sviluppo, Milano, Lombardia, 20133, Italy|Hyogo Medical University Hospital, Hyogo, 663-8501, Japan|Szpital Gdanskiego Uniwersytetu Medycznego; Clinic of developmental neurology, Gda?sk, 80-952, Poland|The Children?s Memorial Health Institute Department of Neurology and Epileptology, Warszawa, 04-730, Poland|Russian Children Neuromuscular Center of Veltischev, Moscow, Moskovskaja Oblast, 125412, Russian Federation|King Faisal Specialist Hospital and Research Centre Building, Riyadh, 11211, Saudi Arabia|Institute for Mother and Child Dr. Vukan Cupic, Belgrade, 11000, Serbia|Hospital Sant Joan De Deu, Esplugues De Llobregas, Barcelona, 08950, Spain|Hospital Universitari Vall d'Hebron; Area Genética Clínica y Molecular, Barcelona, 08035, Spain|Hospital Universitario Virgen del Rocio, Sevilla, 41013, Spain|Universit?ts-Kinderspital (UKBB) Neurop?diatrie, Basel, 4005, Switzerland|Hacettepe University, School of Medicine; Pediatrics Department; Pediatrics Child Neurology Unit, Ankara, 06100, Turkey|Hospital Yeditepe University Kozyatagi; Pediatry, Atasehir- Istanbul, 34752, Turkey|Ams of Ukraine; Inst. of Neurology, Psychiatry & Narcology, Kharkov, 61068, Ukraine Less <<
NCT03032172 Spinal Muscular Atrophy PHASE2 ACTIVE_NOT_RECRUITING 2025-02-25 Stanford University Medical Ce... More >>nter, Palo Alto, California, 94304, United States|Nemours Children's Hospital, Orlando, Florida, 32827, United States|Boston Childrens Hospital, Boston, Massachusetts, 02115, United States|Columbia University Medical Center, New York, New York, 10032, United States|UZ Gent, Gent, 9000, Belgium|UZ Leuven Gasthuisberg, Leuven, 3000, Belgium|Hopital Femme Mere Enfant, Bron, 69677, France|Hopital Roger Salengro, Lille, 59037, France|CHRU de Montpellier, Hopital Gui de Chauliac, Montpellier, 34295, France|H?pital Necker-Enfants Malades, Paris, 75015, France|Hopital des Enfants, Toulouse, 31059, France|Universit?tsklinikum Essen, Essen, 45147, Germany|Universit?tsklinikum Freiburg, Freiburg, 79106, Germany|IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Lazio, 00165, Italy|Policlinico Agostino Gemelli, Roma, Lazio, 00168, Italy|IRCCS Istituto Giannina Gaslini, Genova, Liguria, 16147, Italy|Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano, Lombardia, 20122, Italy|UOSD Malattie Neurodegenerative, Messina, Sicilia, 98125, Italy|UMC Utrecht, Utrecht, 3584 CX, Netherlands|Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie, Warszawa, 02-097, Poland|Universit?ts-Kinderspital (UKBB) Neurop?diatrie, Basel, 4005, Switzerland|Birmingham Heartlands Hospital, Birmingham, B9 5SS, United Kingdom|UCL Institute of Child Health & Great Ormond Street Hospital for Children, London, WC1N 1EH, United Kingdom|The Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, NE1 4LP, United Kingdom Less <<
NCT04256265 Muscular Atrophy, Spinal APPROVED_FOR_MARKETING - Arkansas Children's Hospital; ... More >>Pediatrics, Little Rock, Arkansas, 72202, United States|Children's Hospital Los Angeles, Los Angeles, California, 90010, United States|Stanford University, Palo Alto, California, 94304, United States|University of Colorado in Denver-Anschutz Medical Campus, Aurora, Colorado, 80045, United States|Nemours Children's Hospital, Orlando, Florida, 32827, United States|Comprehensive NeuroBehavioral Institute, Plantation, Florida, 33317, United States|Rare Disease Research, LLC, Atlanta, Georgia, 30318, United States|Ann and Robert H. Lurie Children Hospital of Chicago, Chicago, Illinois, 60611, United States|Southern Illinois University, School of Medicine, Springfield, Illinois, 62702, United States|Indiana Hemophilia & Thrombosis center, Indianapolis, Indiana, 46260, United States|University of Iowa, Iowa City, Iowa, 52242, United States|University of Kansas Medical Center, Kansas City, Kansas, 66160, United States|University of Louisville, Louisville, Kentucky, 40202, United States|Massachusetts General Hospital; Neurology, Boston, Massachusetts, 02114, United States|Helen DeVos Children's Hospital at Spectrum Health, Grand Rapids, Michigan, 49503, United States|University of Mississippi Medical Center; Neurology, Jackson, Michigan, 39216, United States|Gillette Spcl Children's Clin; Pediatric Endocrinology, Saint Paul, Minnesota, 55101, United States|St. Louis Children Hospital, Saint Louis, Missouri, 63110, United States|Goryeb Children's Hospital, Morristown, New Jersey, 07960, United States|Northwell Hospital, New Hyde Park, New York, 11042, United States|NYU Langone, New York, New York, 10003, United States|University of Rochester Medical Center, Rochester, New York, 14642, United States|Wake Forest Baptist Medical Center, Winston-Salem, North Carolina, 27157, United States|Akron Childrens Hospital, Akron, Ohio, 44308, United States|Nationwide Children's Hospital, Columbus, Ohio, 43205, United States|University of Virginia Children's Hospital; Developmental, Charlottesville, Virginia, 22903, United States|University of Wisconsin American Family; Childrens Hospital, Madison, Wisconsin, 53792, United States|Childrens Hospital of Wisconsin, Milwaukee, Wisconsin, 53201, United States|Medical College of Wisconsin, Inc., Milwaukee, Wisconsin, 53226-3596, United States Less <<
NCT05808764 Muscular Atrophy, Spinal PHASE2 RECRUITING 2025-08-31 Ann and Robert H. Lurie Childr... More >>en Hospital of Chicago, Chicago, Illinois, 60611, United States|University Of Michigan, Ann Arbor, Michigan, 48109, United States|Clinic for Special Children., Gordonville, Pennsylvania, 17529, United States|Hopital Universitaire des Enfants Reine Fabiola, Bruxelles, 1020, Belgium|CHR Citadelle, Liege, 4000, Belgium|Children'S Hospital of Eastern Ontario, Ottawa, Ontario, K1H 8L1, Canada|Universitatsklinikum Essen, Essen, 45147, Germany|Fondazione Serena Onlus - CENTRO CLINICO NEMO, Milano, Emilia-Romagna, 20162, Italy|Fondazione Policlinico Univeristario A. Gemelli, Roma, Emilia-Romagna, 00168, Italy|UMC Utrecht, Utrecht, 3508, Netherlands|OUS (Oslo University Hospital), Rikshospitalet, Oslo, 0372, Norway|Uniwersyteckie Centrum Kliniczne, Gda?sk, 80-952, Poland|Instytut Pomnik - Centrum Zdrowia Dziecka, Warszawa, 04-730, Poland Less <<
NCT03779334 Muscular Atrophy, Spinal PHASE2 ACTIVE_NOT_RECRUITING 2027-03-31 Nemours Children's Hospital, O... More >>rlando, Florida, 32827, United States|Sydney Children's Hospital; CENTRE FOR CHILD HEALTH RESEARCH & INNOVATION (CHeRI), Randwick, New South Wales, 2031, Australia|Chr de La Citadelle, Liège, 4000, Belgium|Hospital das Clinicas - FMUSP_X; Neurologia, Sao Paulo, SP, 05403-000, Brazil|Szpital Gdanskiego Uniwersytetu Medycznego; Clinic of developmental neurology, Gda?sk, 80-952, Poland|Russian Children Neuromuscular Center of Veltischev, Moscow, Moskovskaja Oblast, 125412, Russian Federation|Kaohsiung Medical University Chung-Ho Hospital; Pediatric Neurology, Kaohsiung, 807, Taiwan Less <<
NCT05522361 Spinal Muscular Atrophy PHASE4 ACTIVE_NOT_RECRUITING 2026-06-15 Clinic for Special Children, S... More >>trasburg, Pennsylvania, 17579, United States Less <<
NCT05219487 Spinal Muscular Atrophy RECRUITING 2025-12-31 The Ohio State University Wexn... More >>er Medical Center, Columbus, Ohio, 43210, United States Less <<
NCT03920865 Muscular Atrophy, Spinal PHASE1 COMPLETED 2020-01-02 Clinical Pharmacology of Miami... More >>, Inc., Miami, Florida, 33014, United States|Orlando Clinical Research Center, Orlando, Florida, 32809, United States|American Research Corporation Inc., San Antonio, Texas, 78215, United States Less <<

Protocol

Bio Calculators
Preparing Stock Solutions 1mg 5mg 10mg

1 mM

5 mM

10 mM

2.49mL

0.50mL

0.25mL

12.45mL

2.49mL

1.25mL

24.91mL

4.98mL

2.49mL

References

 

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