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Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Sponsor: Novartis Pharmaceuticals
Summary
Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an autosomal recessive, early childhood disease with an incidence of 1:10,000 live births. SMA is the leading cause of infant mortality due to genetic diseases. The purpose of this registry is to assess the long term outcomes of patients with SMA in the context of advances in treatment options and also to characterize and assess long-term safety and effectiveness of OAV-101.
Official title: A Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
700
Start Date
2018-09-25
Completion Date
2038-06-30
Last Updated
2025-01-31
Healthy Volunteers
No
Conditions
Interventions
Prospective observational registry
This prospective observational registry will assess long-term outcomes of patients with a diagnosis of SMA.
Zolgensma
Zolgensma will be given to patients as per normal clinical practice and clinical care will not be mandated by the protocol. As such, the decision to prescribe Zolgensma is separate from the decision to include the patient in this study
Locations (99)
Phoenix Children's Hospital
Phoenix, Arizona, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Loma Linda University Health
Loma Linda, California, United States
Children's Hospital of Los Angeles
Los Angeles, California, United States
University of California Los Angeles Health
Los Angeles, California, United States
Valley Children's Healthcare
Madera, California, United States
Children's Hospital of Orange County
Madera, California, United States
University of California Davis Health System
Sacramento, California, United States
Rady Children's Hospital San Diego
San Diego, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Connecticut Children's Medical Center
Farmington, Connecticut, United States
Yale-New Haven Health System
New Haven, Connecticut, United States
Golisano Children's Hospital of Southwest Florida
Fort Myers, Florida, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Adventist Health System - Florida
Orlando, Florida, United States
Jackson South Medical Center
Palmetto Bay, Florida, United States
Indiana University Health University Hospital (IUHUH)
Indianapolis, Indiana, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
University of Louisville
Louisville, Kentucky, United States
University of Minnesota
Minneapolis, Minnesota, United States
University of Missouri Health System
Columbia, Missouri, United States
Washington University School of Medicine in St. Louis
St Louis, Missouri, United States
Atlantic Health System
Morristown, New Jersey, United States
University of Rochester Medical Center
Rochester, New York, United States
The State University of New York
Stony Brook, New York, United States
Duke Health
Durham, North Carolina, United States
Akron Children's Hospital
Akron, Ohio, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
University Hospitals
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Oklahoma University Medical Center
Oklahoma City, Oklahoma, United States
Oregon Health and Science University
Portland, Oregon, United States
Penn State Hershey
Hershey, Pennsylvania, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
Prisma Health
Greenville, South Carolina, United States
Methodist Le Bonheur Healthcare
Memphis, Tennessee, United States
Child Neurology Consultants of Austin
Austin, Texas, United States
Children's Health
Dallas, Texas, United States
Cook Children's
Fort Worth, Texas, United States
Texas Children's Hospital
Houston, Texas, United States
CHRISTUS Health
San Antonio, Texas, United States
University of Utah
Salt Lake City, Utah, United States
University of Virginia Health System
Charlottesville, Virginia, United States
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States
Virginia Commonwealth University Health System
Richmond, Virginia, United States
Seattle Children's
Seattle, Washington, United States
MultiCare Health System
Tacoma, Washington, United States
University of Wisconsin
Madison, Wisconsin, United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States
University General Hospital Attikon
Chaïdári, Athens, Greece
Penteli Children's Hospital
Pentéli, Attikis, Greece
St. Sophia Children's Hospital
Thessaloniki, Greece
General Hospital of Thessaloniki Ippokrateio
Thessaloniki, Greece
Children's University Hospital-UCD School of Medicine Scoil an Leighis
Dublin, Ireland
Soroka Medical Centre
Beersheba, Israel
Wolfson Medical Center
Holon, Israel
Tel-Aviv Sourasky Medical Center
Holon, Israel
Schneider- Children's Medical Center
Petah Tikva, Israel
Aichi Medical University Hospital
Nagakute-shi, Aichi-ken, Japan
Fujita Health University Hospital
Toyoake-shi, Aichi-ken, Japan
Chiba children's Hospital
Chiba, Chiba, Japan
Kurume University Hospital
Kurume-shi, Fukuoka, Japan
Gifu Prefectural General Medical Center
Gifu, Gifu, Japan
Sapporo Medical University Hospital
Sapporo, Hokkaido, Japan
Kagawa University Hospital
Kita-gun, Kagawa-ken, Japan
Kanagawa Children's Medical Center
Yokohama, Kanagawa, Japan
Kumamoto University Hospital
Kumamoto, Kumamoto, Japan
Miyagi Children's Hospital
Sendai, Miyagi, Japan
Obihiro Kosei Hospital
Hokkaido, Obihiro-shi, Japan
Shiga Medical Center for Children
Moriyama-shi, Shiga, Japan
Hamamatsu University School of Medicine, University Hospital
Hamamatsu, Shizuoka, Japan
Jichi Medical University Hospital
Shimotsuke-shi, Tochigi, Japan
Tokyo Medical University Hospital
Shinjuku-ku, Tokyo, Japan
Center Hospital of the National Center for Global Health and Medicine
Shinjuku-ku, Tokyo, Japan
Fukui Prefectural Hospital
Fukui, Japan
Nikko Memorial Hospital
Hokkaido, Japan
Dokkyo Medical University Saitama Medical Center
Saitama, Japan
Uniwersytecki Szpital Dzieciec
Lublin, Poland
CHUC - Hospital Pediatrico
Coimbra, Portugal
Centro Hospitalar Universitaria de Lisboa Central, EPE
Lisbon, Portugal
Centro Hospitalar Universitario Lisboa Norte, EPE
Lisbon, Portugal
Centro Hospitalar Universitario de Porto, EPE
Porto, Portugal
Centro Hospitalar Universitario de Sao Joao, EPE
Porto, Portugal
Centrul de Recuperare pentru Copii dr. Nicolae Robanescu
Bucharest, Romania
Spitalul Clinic de Psihiatrie
Bucharest, Romania
Almazov National Medical Research Centre
Saint Petersburg, Russia
Kyungpook National University Hospital
Daegu, South Korea
Seoul National University Bundang Hospital
Seongnam-si, South Korea
Seoul National University Children's Hospital
Seoul, South Korea
Severance Hospital
Seoul, South Korea
Samsung Medical Centre
Seoul, South Korea
Pusan National University Yangsan Hospital
Yangsan, South Korea
Yongin Severance Hospital
Yongin-si, South Korea
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, Taiwan
China Medical University Hospital
Taichung, Taiwan
National Taiwan University Hospital
Taipei, Taiwan
Taipei Veterans General Hospital
Taipei, Taiwan
Chang Gung Memorial Hospital Linkou Branch
Taoyuan District, Taiwan