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.
Spinal Muscular Atrophy (SMA)
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.
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
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Phoenix Children's Hospital, Phoenix, Arizona, United States, 85016
Arkansas Children's Hospital, Little Rock, Arkansas, United States, 72202
Loma Linda University Health, Loma Linda, California, United States, 92350
Children's Hospital of Los Angeles, Los Angeles, California, United States, 90027
University of California Los Angeles Health, Los Angeles, California, United States, 90095
Valley Children's Healthcare, Madera, California, United States, 93636
Children's Hospital of Orange County, Madera, California, United States, 93638
University of California Davis Health System, Sacramento, California, United States, 95817
Rady Children's Hospital San Diego, San Diego, California, United States, 92123
Children's Hospital Colorado, Aurora, Colorado, United States, 80045
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Novartis Pharmaceuticals,
Novartis Pharmaceuticals, STUDY_DIRECTOR, Novartis Pharmaceuticals
2038-06-30