Phase
Condition
Muscular Dystrophy
Myasthenia Gravis (Chronic Weakness)
Spinal Muscular Atrophy
Treatment
Prospective observational registry
Zolgensma
Clinical Study ID
All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Patients treated with OAV-101 with a genetically confirmed diagnosis of SMAregardless of the date of diagnosis.
Appropriate consent/assent has been obtained for participation in the registry
Exclusion
Exclusion Criteria:
- Currently enrolled in an interventional clinical trial involving an investigationalmedicinal product to treat SMA.
Note: Patients who are participating in a Compassionate Use Program (CUP) for OAV-101 (Zolgensma) such as a Managed Access Program (MAP), an Expanded Access Program (EAP), Single Patient Investigational New Drug (IND) (SPI) or Named Patient Program (NPP) are eligible to enroll in the registry regardless of the date of a genetic or clinical diagnosis of SMA.
Study Design
Study Description
Connect with a study center
University General Hospital Attikon
Chaïdári, Athens 124 62
GreeceActive - Recruiting
Penteli Children's Hospital
Penteli, Attikis 152 36
GreeceActive - Recruiting
St. Sophia Children's Hospital
Thessaloniki, 115 27
GreeceActive - Recruiting
General Hospital of Thessaloniki Ippokrateio
Thessaloníki, 546 42
GreeceActive - Recruiting
Children's University Hospital-UCD School of Medicine Scoil an Leighis
Dublin, 1 D01 XD99
IrelandActive - Recruiting
Soroka Medical Centre
Be'er Sheva, 85025
IsraelActive - Recruiting
Wolfson Medical Center
Holon, 5822012
IsraelActive - Recruiting
Tel-Aviv Sourasky Medical Center
H̱olon, 582201
IsraelActive - Recruiting
Schneider- Children's Medical Center
Petah tikva, 4920235
IsraelActive - Recruiting
Konan Kosei Hospital
Konan, Aichi 483-8704
JapanSite Not Available
Aichi Medical University Hospital
Nagakute-shi, Aichi 480-1195
JapanSite Not Available
Fujita Health University Hospital
Toyoake-shi, Aichi 470-1192
JapanSite Not Available
Chiba children's Hospital
Chiba-shi, Chiba 266-0007
JapanSite Not Available
Ehime University Hospital
Toon, Ehime 791-0295
JapanSite Not Available
Kurume University Hospital
Kurume-shi, Fukuoka 830-0011
JapanSite Not Available
Gifu Prefectural General Medical Center
Gifu-shi, Gifu 500-8717
JapanSite Not Available
Nikko Memorial Hospital
Muroran-shi, Hokkaido 051-8501
JapanSite Not Available
Obihiro Kosei Hospital
Obihiro, Hokkaido 080-0024
JapanSite Not Available
Sapporo Medical University Hospital
Sapporo-shi, Hokkaido 060-8543
JapanSite Not Available
Kagawa University Hospital
Kita-gun, Kagawa 761-0793
JapanSite Not Available
Kanagawa Children's Medical Center
Yokohama-shi, Kanagawa 232-8555
JapanSite Not Available
Kawasaki Municipal Tama Hospital
Kanagawa, Kawasaki-shi 214-8525
JapanSite Not Available
Kagawa University Hospital
Kagawa, Kita-gun 761-0793
JapanSite Not Available
Kumamoto University Hospital
Kumamoto-shi, Kumamoto 860-8556
JapanSite Not Available
Miyagi Children's Hospital
Sendai-shi, Miyagi 989-3126
JapanSite Not Available
Obihiro Kosei Hospital
Hokkaido, Obihiro-shi 080-0024
JapanSite Not Available
Dokkyo Medical University
Koshigaya, Saitama 343-8555
JapanSite Not Available
Shiga Medical Center for Children
Moriyama-shi, Shiga 524-0022
JapanSite Not Available
Hamamatsu University School of Medicine, University Hospital
Hamamatsu-shi, Shizuoka 431-3192
JapanSite Not Available
Jichi Medical University Hospital
Shimotsuke-shi, Tochigi 329-0498
JapanSite Not Available
Tokyo Metropolitan Neurological Hospital
Fuchu, Tokyo 183-0042
JapanSite Not Available
Center Hospital of the National Center for Global Health and Medicine
Shinjuku-ku, Tokyo 162-8655
JapanSite Not Available
Tokyo Medical University Hospital
Shinjuku-ku, Tokyo 160-0023
JapanSite Not Available
Chiba children's Hospital
Chiba, 266-0007
JapanSite Not Available
Fukui Prefectural Hospital
Fukui, 910-8526
JapanSite Not Available
Gifu Prefectural General Medical Center
Gifu, 500-8717
JapanSite Not Available
Nikko Memorial Hospital
Hokkaido, 051-0005
JapanSite Not Available
Dokkyo Medical University Saitama Medical Center
Saitama, 343-0845
JapanSite Not Available
Saitama Children's Medical Center
Saitama, 330-8777
JapanSite Not Available
Keio University Hospital
Tokyo, 160-8582
JapanSite Not Available
Kyungpook National University Hospital
Daegu, 41944
Korea, Republic ofActive - Recruiting
Seoul National University Bundang Hospital
Seongnam-si, 13620
Korea, Republic ofActive - Recruiting
Samsung Medical Centre
Seoul, 06351
Korea, Republic ofActive - Recruiting
Seoul National University Children's Hospital
Seoul, 03080
Korea, Republic ofActive - Recruiting
Severance Hospital
Seoul, 03722
Korea, Republic ofActive - Recruiting
Pusan National University Yangsan Hospital
Yangsan, 50612
Korea, Republic ofActive - Recruiting
Yongin Severance Hospital
Yongin-si, 16995
Korea, Republic ofActive - Recruiting
Uniwersytecki Szpital Dzieciec
Lublin, 20-093
PolandActive - Recruiting
CHUC - Hospital Pediatrico
Coimbra, 300-6020
PortugalActive - Recruiting
Centro Hospitalar Universitaria de Lisboa Central, EPE
Lisboa, 1169-045
PortugalActive - Recruiting
Centro Hospitalar Universitario Lisboa Norte, EPE
Lisboa, 1649-035
PortugalActive - Recruiting
Centro Hospitalar Universitario de Porto, EPE
Porto, 4050-371
PortugalActive - Recruiting
Centro Hospitalar Universitario de Sao Joao, EPE
Porto, 4200-319
PortugalActive - Recruiting
Centrul de Recuperare pentru Copii dr. Nicolae Robanescu
Bucharest, 041408
RomaniaActive - Recruiting
Spitalul Clinic de Psihiatrie
Bucharest, 041914
RomaniaActive - Recruiting
Almazov National Medical Research Centre
Saint Petersburg, 197341
Russian FederationActive - Recruiting
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung city, 80756
TaiwanActive - Recruiting
China Medical University Hospital
Taichung, 404327
TaiwanActive - Recruiting
National Taiwan University Hospital
Taipei, 100229
TaiwanActive - Recruiting
Taipei Veterans General Hospital
Taipei, 11217
TaiwanActive - Recruiting
Chang Gung Memorial Hospital Linkou Branch
Taoyuan, 333
TaiwanActive - Recruiting
Phoenix Children's Hospital
Phoenix, Arizona 85016
United StatesActive - Recruiting
Arkansas Children's Hospital
Little Rock, Arkansas 72202
United StatesActive - Recruiting
Loma Linda University Health
Loma Linda, California 92350
United StatesCompleted
Children's Hospital of Los Angeles
Los Angeles, California 90027
United StatesActive - Recruiting
University of California Los Angeles Health
Los Angeles, California 90095
United StatesActive - Recruiting
Children's Hospital of Orange County
Madera, California 93638
United StatesActive - Recruiting
Valley Children's Healthcare
Madera, California 93636
United StatesActive - Recruiting
Children's Hospital of Orange County
Orange, California 92868-3874
United StatesSite Not Available
University of California Davis Health System
Sacramento, California 95817
United StatesActive - Recruiting
Rady Children's Hospital San Diego
San Diego, California 92123
United StatesActive - Recruiting
Children's Hospital Colorado
Aurora, Colorado 80045
United StatesActive - Recruiting
Connecticut Children's Medical Center
Farmington, Connecticut 06034
United StatesActive - Recruiting
Yale-New Haven Health System
New Haven, Connecticut 06510
United StatesActive - Recruiting
Golisano Children's Hospital of Southwest Florida
Fort Myers, Florida 33908
United StatesActive - Recruiting
Nicklaus Children's Hospital
Miami, Florida 33155
United StatesCompleted
Adventist Health System - Florida
Orlando, Florida 32803
United StatesCompleted
Jackson South Medical Center
Palmetto Bay, Florida 33157
United StatesActive - Recruiting
Indiana University Health University Hospital (IUHUH)
Indianapolis, Indiana 46202
United StatesActive - Recruiting
University of Iowa
Iowa City, Iowa 52242-1009
United StatesActive - Recruiting
University of Kansas Medical Center
Kansas City, Kansas 66160
United StatesActive - Recruiting
University of Louisville
Louisville, Kentucky 40202
United StatesActive - Recruiting
Boston Children's Hospital
Boston, Massachusetts 02115
United StatesSite Not Available
Beaumont Health System
Royal Oak, Michigan 48073
United StatesSite Not Available
University of Minnesota
Minneapolis, Minnesota 55455
United StatesActive - Recruiting
University of Missouri Health System
Columbia, Missouri 65212
United StatesActive - Recruiting
Washington University School of Medicine in St. Louis
Saint Louis, Missouri 63110
United StatesActive - Recruiting
Atlantic Health System
Morristown, New Jersey 07960
United StatesActive - Recruiting
University of Rochester Medical Center
Rochester, New York 14642
United StatesActive - Recruiting
The State University of New York
Stony Brook, New York 11794
United StatesActive - Recruiting
Duke Health
Durham, North Carolina 27705
United StatesActive - Recruiting
Akron Children's Hospital
Akron, Ohio 44308-1062
United StatesCompleted
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229-3026
United StatesActive - Recruiting
University Hospitals
Cleveland, Ohio 44106
United StatesCompleted
Nationwide Children's Hospital
Columbus, Ohio 43205
United StatesActive - Recruiting
Oklahoma University Medical Center
Oklahoma City, Oklahoma 73104
United StatesCompleted
Oregon Health and Science University
Portland, Oregon 97239-3098
United StatesActive - Recruiting
Penn State Hershey
Hershey, Pennsylvania 17033
United StatesActive - Recruiting
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania 15224
United StatesActive - Recruiting
Prisma Health
Greenville, South Carolina 29615
United StatesActive - Recruiting
Methodist Le Bonheur Healthcare
Memphis, Tennessee 38103
United StatesActive - Recruiting
Child Neurology Consultants of Austin
Austin, Texas 78731
United StatesCompleted
Children's Health
Dallas, Texas 75207
United StatesCompleted
Cook Children's
Fort Worth, Texas 76104
United StatesActive - Recruiting
Texas Children's Hospital
Houston, Texas 77030
United StatesActive - Recruiting
CHRISTUS Health
San Antonio, Texas 78207
United StatesActive - Recruiting
University of Utah
Salt Lake City, Utah 84113
United StatesActive - Recruiting
University of Virginia Health System
Charlottesville, Virginia 22908
United StatesActive - Recruiting
Children's Hospital of The King's Daughters
Norfolk, Virginia 23507
United StatesActive - Recruiting
Virginia Commonwealth University Health System
Richmond, Virginia 23298
United StatesActive - Recruiting
Seattle Children's
Seattle, Washington 98105
United StatesActive - Recruiting
MultiCare Health System
Tacoma, Washington 98405
United StatesActive - Recruiting
University of Wisconsin
Madison, Wisconsin 53792
United StatesActive - Recruiting
Children's Hospital of Wisconsin
Milwaukee, Wisconsin 53226
United StatesActive - Recruiting
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