EICHORCondition: Spinal Muscular Atrophy (SMA) · Sponsor: Novartis Pharmaceuticals
Sponsor, CRO or site team? See where this study is running → — listed sites, countries and recruiting context. Everything below is written for patients and caregivers.
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 description comes directly from the study's public registry record.
Novartis Gene Therapies · 1-888-669-6682 · novartis.email@novartis.com
Novartis Gene Therapies · +4161324111
Always discuss trial participation with your own doctor first.
| Phoenix Children's Hospital | Phoenix, Arizona, United States | Recruiting |
| Arkansas Children's Hospital | Little Rock, Arkansas, United States | Recruiting |
| Loma Linda University Health | Loma Linda, California, United States | Completed |
| Children's Hospital of Los Angeles | Los Angeles, California, United States | Recruiting |
| University of California Los Angeles Health | Los Angeles, California, United States | Recruiting |
| Valley Children's Healthcare | Madera, California, United States | Recruiting |
| Children's Hospital of Orange County | Madera, California, United States | Recruiting |
| University of California Davis Health System | Sacramento, California, United States | Recruiting |
| Rady Children's Hospital San Diego | San Diego, California, United States | Recruiting |
| Children's Hospital Colorado | Aurora, Colorado, United States | Recruiting |
| Connecticut Children's Medical Center | Farmington, Connecticut, United States | Recruiting |
| Yale-New Haven Health System | New Haven, Connecticut, United States | Recruiting |
+ 87 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT04174157