Can Ficicioglu , Elizabeth Berry-Kravis , Walla Al-Hertani , Raymond Y. Wang , Marc Patterson , Loren Pena , Kristina Julich , Damara Ortiz , Paula Schleifer , Paul Hillman , Caroline A. Hastings , Blair Orr , Ronan J. O'Reilly , Daniel Gallo
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Arimoclomol safety profile in the treatment of Niemann-Pick disease type C in a real-world setting: Long-term safety data from an expanded access program in the United States
期刊介绍:
Molecular Genetics and Metabolism contributes to the understanding of the metabolic and molecular basis of disease. This peer reviewed journal publishes articles describing investigations that use the tools of biochemical genetics and molecular genetics for studies of normal and disease states in humans and animal models.