Work

Genetic Replacement Therapy (GRT) Clinical Trial with AVXS-101 in Presymptomatic Spinal Muscular Atrophy (SMA): Design of the Phase III Trial and Baseline Demographic Data

Year: 2019

Type: conference-paper

Source: Nervenheilkunde

DOI: 10.1055/s-0039-1685081

Authors: M Schultz, K Swoboda, E Kernbauer, F Ogrinc, DE Feltner, BE McGill, DM Sproule, KA Strauss

Institutions: University of Wisconsin–Madison, University of Wisconsin Health, Massachusetts General Hospital

Language: English

Topic: Neurogenetic and Muscular Disorders Research

Subfield: Genetics

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W2952114962

FWCI:

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