Study Description
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. This is a prospective, multi center, multinational, non-interventional observational study. All patients will be managed according to the clinical site's normal clinical practice, i.e., the diagnostic and clinical treatment/practice process that a clinician chooses according to their clinical judgement for an SMA patient. Clinical care will not be driven by the protocol. No additional visits or investigations will be performed beyond normal clinical practice. Patients will be followed for 15 years from enrolment or until death, whichever is sooner.
Interventions
Prospective observational registry
Zolgensma
Eligibility Criteria
Inclusion Criteria:
* Patients treated with OAV-101 with a genetically confirmed diagnosis of SMA regardless of the date of diagnosis.
* Appropriate consent/assent has been obtained for participation in the registry
Exclusion Criteria:
- Currently enrolled in an interventional clinical trial involving an investigational medicinal 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.
General Hospital of Thessaloniki Ippokrateio
Recruiting
Thessaloníki,546 42,Greece
Dimitrios Zafeiriou, MD
Penteli Children's Hospital
Recruiting
Penteli,Attikis,152 36,Greece
Chrysanthi Tsimakidi
St. Sophia Children's Hospital
Recruiting
Thessaloniki,115 27,Greece
Maria Roser-Pons, MD, MD
University General Hospital Attikon
Recruiting
Chaïdári,Athens,124 62,Greece
Argyrios Dinopoulos, MD
Children's University Hospital-UCD School of Medicine Scoil an Leighis
Recruiting
Dublin,1 d01 xd99,Ireland
Tel-Aviv Sourasky Medical Center
Recruiting
H̱olon,582201,Israel
Aviva Fattal, MD
Schneider- Children's Medical Center
Recruiting
Petah tikva,4920235,Israel
Sharon Aharoni, MD
Soroka Medical Centre
Recruiting
Be'er Sheva,85025,Israel
Iris Noyman, MD
Wolfson Medical Center
Recruiting
Holon,5822012,Israel
Mira Ginsberg, MD
Seoul National University Children's Hospital
Recruiting
Seoul,03080,Korea, Republic of
Jong Hee Chae
MD
Seoul National University Bundang Hospital
Recruiting
Seongnam-si,13620,Korea, Republic of
Anna Cho, MD
Samsung Medical Centre
Recruiting
Seoul,06351,Korea, Republic of
Jiwon Lee
Severance Hospital
Recruiting
Seoul,03722,Korea, Republic of
Hoonchul Kang, MD
Yongin Severance Hospital
Recruiting
Yongin-si,16995,Korea, Republic of
Haneul Lee
Pusan National University Yangsan Hospital
Recruiting
Yangsan,50612,Korea, Republic of
Yunjin Lee, MD
Kyungpook National University Hospital
Recruiting
Daegu,41944,Korea, Republic of
Yunjeong Lee, MD
Uniwersytecki Szpital Dzieciec
Recruiting
Lublin,20-093,Poland
Magdalena Chroscinska-Krawczyk, MD
Centro Hospitalar Universitario de Sao Joao, EPE
Recruiting
Porto,4200-319,Portugal
Raquel Carvalho Sousa
CHUC - Hospital Pediatrico
Recruiting
Coimbra,300-6020,Portugal
Joana Ribeiro, MD
Centro Hospitalar Universitario Lisboa Norte, EPE
Recruiting
Lisboa,1649-035,Portugal
Teresa Moreno, MD
Centro Hospitalar Universitaria de Lisboa Central, EPE
Recruiting
Lisboa,1169-045,Portugal
Jose Pedro Vieira, MD
Centro Hospitalar Universitario de Porto, EPE
Recruiting
Porto,4050-371,Portugal
Cristina Garrido, MD
Centrul de Recuperare pentru Copii dr. Nicolae Robanescu
Recruiting
Bucharest,041408,Romania
Madalina Cristina Leanca
Spitalul Clinic de Psihiatrie
Recruiting
Bucharest,041914,Romania
Loana Minciu
Almazov National Medical Research Centre
Recruiting
Saint Petersburg,197341,Russian Federation
Natalia Petrova, md
Chang Gung Memorial Hospital Linkou Branch
Recruiting
Taoyuan,333,Taiwan
I-Jun CHOU, MD
China Medical University Hospital
Recruiting
Taichung,404327,Taiwan
I-Ching Chou
National Taiwan University Hospital
Recruiting
Taipei,100229,Taiwan
Yin-Hsiu CHIEN, MD
Taipei Veterans General Hospital
Recruiting
Taipei,11217,Taiwan
Dau-Ming NIU
Kaohsiung Medical University Chung-Ho Memorial Hospital
Recruiting
Kaohsiung city,80756,Taiwan
Yuh-Jyh Jong, MD
University of Pittsburgh Medical Center
Recruiting
Pittsburgh,Pennsylvania,15224,United States
Hoda Abdel-Hamid, MD
Arkansas Children's Hospital
Recruiting
Little Rock,Arkansas,72202,United States
Kapil Arya, MD
University of Iowa
Recruiting
Iowa City,Iowa,52242-1009,United States
Katherine Mathews, MD
University of Utah
Recruiting
Salt Lake City,Utah,84113,United States
Russell Butterfield, MD, PhD
University of California Davis Health System
Recruiting
Sacramento,California,95817,United States
Nanette Joyce, DO
Atlantic Health System
Recruiting
Morristown,New Jersey,07960,United States
Darius Adams, MD
Nationwide Children's Hospital
Recruiting
Columbus,Ohio,43205,United States
Megan Waldrop, MD
Children's Hospital of Wisconsin
Recruiting
Milwaukee,Wisconsin,53226,United States
Matthew Harmelink, MD
Golisano Children's Hospital of Southwest Florida
Recruiting
Fort Myers,Florida,33908,United States
Britt Stroud, MD
Cook Children's
Recruiting
Fort Worth,Texas,76104,United States
Warren Marks, MD
Valley Children's Healthcare
Recruiting
Madera,California,93636,United States
Raymund David, MD
University of Minnesota
Recruiting
Minneapolis,Minnesota,55455,United States
Peter Karachunski, MD
Seattle Children's
Recruiting
Seattle,Washington,98105,United States
Seth Perlman, MD
Connecticut Children's Medical Center
Recruiting
Farmington,Connecticut,06034,United States
Gyula Acsadi, MD, PhD
Duke Health
Recruiting
Durham,North Carolina,27705,United States
Mai Elmallah, MD
Prisma Health
Recruiting
Greenville,South Carolina,29615,United States
Addie Hunnicutt, MD
University of Virginia Health System
Recruiting
Charlottesville,Virginia,22908,United States
Rebecca Scharf, MD
Rady Children's Hospital San Diego
Recruiting
San Diego,California,92123,United States
Chamindra Konersman, MD
Jackson South Medical Center
Recruiting
Palmetto Bay,Florida,33157,United States
Paula Schleifer, MD
Oregon Health and Science University
Recruiting
Portland,Oregon,97239-3098,United States
Erika Finanger, MD
Texas Children's Hospital
Recruiting
Houston,Texas,77030,United States
Timothy Lotze, MD
Children's Hospital of Los Angeles
Recruiting
Los Angeles,California,90027,United States
Leigh Maria Ramos-Platt, MD
University of Rochester Medical Center
Recruiting
Rochester,New York,14642,United States
Bohoon Lee, MD
Kim Hart Kim_
University of Missouri Health System
Recruiting
Columbia,Missouri,65212,United States
Jane Emerson, MD
Children's Hospital of Orange County
Recruiting
Madera,California,93638,United States
Amanda Fernandez, MD
MultiCare Health System
Recruiting
Tacoma,Washington,98405,United States
Anisha Schwarz, MD
Yale-New Haven Health System
Recruiting
New Haven,Connecticut,06510,United States
Cristian Ionita, MD
Cincinnati Children's Hospital Medical Center
Recruiting
Cincinnati,Ohio,45229-3026,United States
Cuixia Tian, MD
University of Kansas Medical Center
Recruiting
Kansas City,Kansas,66160,United States
Jeffrey Statland, MD
Children's Hospital of The King's Daughters
Recruiting
Norfolk,Virginia,23507,United States
Crystal Proud, MD
Indiana University Health University Hospital (IUHUH)
Recruiting
Indianapolis,Indiana,46202,United States
Marcia Felker, MD
Penn State Hershey
Recruiting
Hershey,Pennsylvania,17033,United States
CHRISTUS Health
Recruiting
San Antonio,Texas,78207,United States
Melissa Svoboda, MD
Methodist Le Bonheur Healthcare
Recruiting
Memphis,Tennessee,38103,United States
Elena Caron, MD
Children's Hospital Colorado
Recruiting
Aurora,Colorado,80045,United States
Julie Parsons, MD
Phoenix Children's Hospital
Recruiting
Phoenix,Arizona,85016,United States
Saunder Bernes, MD
Washington University School of Medicine in St. Louis
Recruiting
Saint Louis,Missouri,63110,United States
Craig Zaidman, MD
University of Wisconsin
Recruiting
Madison,Wisconsin,53792,United States
Jennifer Kwon, MD
University of Louisville
Recruiting
Louisville,Kentucky,40202,United States
Arpita Lakhotia, MD
University of California Los Angeles Health
Recruiting
Los Angeles,California,90095,United States
Perry Shieh, MD, PhD
The State University of New York
Recruiting
Stony Brook,New York,11794,United States
Simona Treidler, MD
Virginia Commonwealth University Health System
Recruiting
Richmond,Virginia,23298,United States
Amy Harper, MD
Worldwide Contacts
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