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Individuals interested in a collaboration or potential position in the lab should reach out to Rebecca at ahrensnicklasr@email.chop.edu.
Members of the Ahrens-Nicklas lab aim to identify, characterize, and develop better treatments for inherited disorders of biochemistry (i.e., inborn errors of metabolism, IEM).
While individually rare, IEM occur in approximately one in 1,500 births. The vast majority of patients have neurologic and/or cardiac symptoms that do not respond to therapy, even if a treatment can improve serum biochemical markers of disease.
Working with genetic, biochemical, and electrophysiologic techniques in cells, model organisms, and human subjects, the lab team investigates the mechanisms that drive neurologic and cardiac dysfunction in patients with IEM. They also evaluate the efficacy of a number of new therapeutic strategies for IEM (including small-molecule, network-directed, and gene therapies).
Current research efforts include:
Assistant Professor of Pediatrics
Dr. Ahrens-Nicklas works to understand why patients with inherited biochemical disorders often suffer severe, untreatable neurologic and cardiac symptoms. She strives to elucidate the link between biochemistry and network excitability, in order to drive new approaches to therapy.