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An Open Label, Single Arm, Dose Escalation Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of GCB-002 in the Treatment of Female Subjects With MECP2 Gene Mutation in Patients With Rett Syndrome
An Open Label, Single Arm IIT Clinical Study Evaluating the Safety, Tolerability, and Preliminary Efficacy of GCB-001 in the Treatment of Patients With Delayed Onset Type 2 SMA Who Can Sit Alone But Cannot Walk.
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)