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1601. Untargeted metabolomic profiling in a patient with glycogen storage disease Ib receiving empagliflozin treatment. Issue 4 (22nd May 2022)

1603. Update on transcobalamin deficiency: clinical presentation, treatment and outcome. Issue 3 (5th December 2013)

1605. Use of an adeno‐associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model. Issue 6 (19th August 2021)

1608. Use of Elamipretide in patients assigned treatment in the compassionate use program: Case series in pediatric patients with rare orphan diseases. Issue 1 (21st September 2022)

1609. Use of sapropterin dihydrochloride in maternal phenylketonuria. A European experience of eight cases. Issue 5 (1st May 2014)