Phase
Condition
Myasthenia Gravis (Chronic Weakness)
Spinal Muscular Atrophy
Muscular Dystrophy
Treatment
nusinersen
Clinical Study ID
All Genders
Study Summary
Eligibility Criteria
Inclusion
Key Inclusion Criteria:
Signed informed consent of parent or guardian and signed informed assent ofparticipant, if indicated per participant's age and institutional guidelines.
Completion of the index study in accordance with the study protocol or as a resultof Sponsor decision (e.g., early termination of the index study) within thepreceding 16 weeks
Exclusion
Key Exclusion Criteria:
Have any condition or worsening condition which in the opinion of the Investigatorwould make the participant unsuitable for enrollment, or could interfere with theparticipant participating in or completing the study
Clinically significant abnormalities in hematology or clinical chemistry parametersor electrocardiogram (ECG), as assessed by the Site Investigator, at the Screeningvisit that would render the participant unsuitable for participation in the study
Participant's parent or legal guardian is not willing or able to meet standard ofcare guidelines (including vaccinations and respiratory syncytial virus prophylaxisif available), nor provide nutritional and respiratory support throughout the study
Treatment with another investigational agent, biological agent, or device within onemonth of Screening, or 5 half-lives of study agent, whichever is longer
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Study Design
Study Description
Connect with a study center
Research site
Sydney, New South Wales 2031
AustraliaSite Not Available
Sydney Children's Hospital Clinical Research Centre
Sydney, New South Wales 2031
AustraliaSite Not Available
Research site
Parkville, Victoria 3052
AustraliaSite Not Available
Royal Children's Hospital
Parkville, Victoria 3052
AustraliaSite Not Available
Research site
Brussels, 1020
BelgiumSite Not Available
Universitair Kinderziekenhuis Koningin Fabiola
Brussels, 1020
BelgiumSite Not Available
BC Children's Hospital / UBC
Vancouver, British Columbia V6H 3V4
CanadaSite Not Available
Research site
Vancouver, British Columbia V6H 3V4
CanadaSite Not Available
Children's Health Research Institute
Brussel, Ontario N6A 5W9
CanadaSite Not Available
Research site
London, Ontario N6A 5W9
CanadaSite Not Available
Hospital for Sick Children
Toronto, Ontario M5G 0A4
CanadaSite Not Available
McGill University Health Centre
Montreal, Quebec H4A 3J1
CanadaSite Not Available
Research site
Montreal, Quebec H4A 3J1
CanadaSite Not Available
Armand Trousseau Hospital, I-Motion
Paris, Paris 9 75012
FranceSite Not Available
Research site
Paris, 75012
FranceSite Not Available
LMU-Campus Innenstadt
Muenchen, Bayern 80337
GermanySite Not Available
Research site
Munich, Bayern 80337
GermanySite Not Available
Research site
Essen, 45147
GermanySite Not Available
Universitatsklinikum Essen
Essen, 45147
GermanySite Not Available
Research site
Freiburg, 79106
GermanySite Not Available
Universitaetsklinikum Freiburg
Freiburg, 79106
GermanySite Not Available
Research site
Hong Kong, Hong Kong SAR 999077
Hong KongSite Not Available
The University of Hong Kong
Hong Kong, Hong Kong SAR 999077
Hong KongSite Not Available
Pediatric Neurology Unit, Catholic University
Essen, 00168
ItalySite Not Available
Istituto Giannina Gaslini, Centro Traslazionale di Miologia
Genova, 16147
ItalySite Not Available
Research site
Genova, 16147
ItalySite Not Available
Department of Neuroscience, Università di Messina, AOU Polic
Messina, 98125
ItalySite Not Available
Research site
Messina, 98125
ItalySite Not Available
Research site
Milan, 20162
ItalySite Not Available
Research site
Rome, 00168
ItalySite Not Available
Aichi Children's Health and Medical Center
Obu, Aichi 474-0038
JapanSite Not Available
Research site
Obu, Aichi 474-0038
JapanSite Not Available
Hyogo College of Medicine
Nishinomiya, Hyogo 663-8501
JapanSite Not Available
Research site
Nishinomiya, Hyogo 663-8501
JapanSite Not Available
Hyogo College of Medicine
Nishinomya-shi, Hyogo
JapanSite Not Available
Research site
Shinjuku-ku, Tokyo 162-8666
JapanSite Not Available
Tokyo Women's Medical University
Shinjuku-ku, Tokyo 162-8666
JapanSite Not Available
Kumamoto University Hospital
Kumamoto, 860-8556
JapanSite Not Available
Research site
Kumamoto, 860-8556
JapanSite Not Available
Miyagi Prefectural Children Hospital
Miyagi, 989-3126
JapanSite Not Available
Research site
Miyazaki, 989-3126
JapanSite Not Available
University of Miyazaki Hospital
Miyazaki, 889-1692
JapanSite Not Available
Research site
Seoul, Korea 3080
Korea, Republic ofSite Not Available
Seoul National University Hospital
Seoul, Korea 3080
Korea, Republic ofSite Not Available
Hospital Sant Joan de Deu
Barcelona, 08950
SpainSite Not Available
Research site
Barcelona, 08950
SpainSite Not Available
Hospital Universitario Vall de Hebron
Hebron, 08035
SpainSite Not Available
Research site
Hebron, 08035
SpainSite Not Available
Hospital Universitario La Paz
Madrid, 28046
SpainSite Not Available
Research site
Madrid, 28046
SpainSite Not Available
Research site
Gothenburg, SE 416 86
SwedenSite Not Available
The Queen Silvia Children's Hospital
Gothenburg, SE 416 86
SwedenSite Not Available
Kaohsiung Medical University Hospital
Kaohsiung City, 807
TaiwanSite Not Available
Research site
Ankara, 6100
TurkeySite Not Available
Uníversity of Hacettepe
Ankara, 6100
TurkeySite Not Available
Marmara University Pendik Training and Research Hospital
Istanbul, 34662
TurkeySite Not Available
Research site
Istanbul, 34662
TurkeySite Not Available
MRC Centre for Neuromuscular Diseases at Newcastle
Newcastle, Northumberland NE1 3BZ
United KingdomSite Not Available
Research site
London, WC1N 1EH
United KingdomSite Not Available
UCL Institute of Child Health
London, WC1N 1EH
United KingdomSite Not Available
Research site
Newcastle, NE1 3BZ
United KingdomSite Not Available
David Geffen School of Medicine at UCLA
Los Angeles, California 90095-8344
United StatesSite Not Available
Research site
Los Angeles, California 90095
United StatesSite Not Available
Research site
Palo Alto, California 94305
United StatesSite Not Available
Stanford University School of Medicine
Palo Alto, California 94305
United StatesSite Not Available
Children's Hospital Colorado
Aurora, Colorado 80045
United StatesSite Not Available
Research site
Aurora, Colorado 80045
United StatesSite Not Available
Connecticut Children's Medical Center
Hartford, Connecticut 06106
United StatesSite Not Available
Research site
Hartford, Connecticut 06106
United StatesSite Not Available
Nemours Children's Clinic
Orlando, Florida 32827
United StatesSite Not Available
Research site
Orlando, Florida 32827
United StatesSite Not Available
Research site
Chicago, Illinois 60611
United StatesSite Not Available
Ann & Robert H. Lurie Children's Hospital of Chicago
New York, Illinois 60611
United StatesSite Not Available
Research Site
Baltimore, Maryland 21287
United StatesSite Not Available
The Johns Hopkins Hospital
Baltimore, Maryland 21287
United StatesSite Not Available
Boston Children's Hospital
Boston, Massachusetts 02115
United StatesSite Not Available
Research site
Boston, Massachusetts 02115
United StatesSite Not Available
Research site
Minneapolis, Minnesota 55101
United StatesSite Not Available
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota 55101
United StatesSite Not Available
Research site
Saint Paul, Minnesota 55101
United StatesSite Not Available
Research site
Saint Louis, Missouri 63110
United StatesSite Not Available
Washington University School of Medicine
Saint Louis, Missouri 63110
United StatesSite Not Available
Washington University School of Medicine
St. Louis, Missouri 63110
United StatesSite Not Available
Columbia University Medical Center
New York, New York 10032
United StatesSite Not Available
Research site
New York, New York 10032
United StatesSite Not Available
Duke University School of Medicine
Durham, North Carolina 27710
United StatesSite Not Available
Research site
Durham, North Carolina 27710
United StatesSite Not Available
Duke University School of Medicine
Miyagi, North Carolina 27710
United StatesSite Not Available
Oregon Health Sciences University
Durham, Oregon 97239
United StatesSite Not Available
Research site
Portland, Oregon 97239
United StatesSite Not Available
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesSite Not Available
Research site
Philadelphia, Pennsylvania 19104
United StatesSite Not Available
Children's Medical Center
Dallas, Texas 75235
United StatesSite Not Available
Research site
Dallas, Texas 75235
United StatesSite Not Available
University of Utah
Obu, Aichi, Utah 84112
United StatesSite Not Available
Research site
Salt Lake City, Utah 84132
United StatesSite Not Available
Research Site
Seattle, Washington 98105
United StatesSite Not Available
Seattle Children's Hospital
Seattle, Washington 98105
United StatesSite Not Available
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