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Effectiveness of Riboflavin in Inherited Metabolic Diseases: A Systematic Review.
Bregje Jaeger1, Nina N Stolwijk2,3, Femke Aaldering4
1Department of Child Neurology, Emma Children's Hospital, Amsterdam UMC, Amsterdam, the Netherlands.
Riboflavin (RF, vitamin B2) is effective for treating MADD type 3, RTD 2,3, ACAD 9, and FAD transporter deficiency in inherited metabolic diseases (IMDs). While safe and beneficial for these conditions, its efficacy remains uncertain for many other IMDs, warranting further investigation.
Area of Science:
- Biochemistry
- Genetics
- Metabolic Disorders
Background:
- Riboflavin (vitamin B2) is essential for cellular processes.
- Its therapeutic role in inherited metabolic diseases (IMDs) is not fully established.
- Investigating RF efficacy and safety in IMDs is crucial for patient management.
Conclusions:
- Riboflavin therapy is safe and effective for MADD type 3, RTD 2 and 3, ACAD9, and FAD transporter deficiency.
- Ensuring patient access to RF for these specific IMDs is critical.
- For IMDs with uncertain RF response, trials with defined outcomes may be considered.
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