Genetic Newborn Screening for Cystinosis and Spinal Muscular Atrophy
Conditions:   Cystinosis;   Cystinosis, Nephropathic;   Spinal Muscular Atrophy Intervention:   Diagnostic Test: molecular-based screening Sponsors:   Cystinose Stiftung;   Labor Becker & Kollegen, Munich, Germany;   Screening Labor Hannover;   Genetikum, Ulm, Germany;   Bavarian State Office of Health and Food Safety (LGL), Unterschleißheim, Germany;   Internal Medicine, Freiburg University Hospital, Freiburg, Germany;   Attorney DSZ Rechtsanwälte GmbH, Barkhovenallee 1, 45239 Essen Completed (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - September 7, 2023 Category: Research Source Type: clinical trials

A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma
Condition:   Spinal Muscular Atrophies Intervention:   Other: Onasemnogene Abeparvovec Sponsor:   Novartis Pharmaceuticals Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - August 31, 2023 Category: Research Source Type: clinical trials