Recently, the safety and efficacy of gene therapy were evaluated in patients with Crigler-Najjar syndrome (CNS). Although it is a promising curative option for CNS, many doubts still persist about… Click to show full abstract
Recently, the safety and efficacy of gene therapy were evaluated in patients with Crigler-Najjar syndrome (CNS). Although it is a promising curative option for CNS, many doubts still persist about its long-term efficacy and safety. Furthermore, there is a risk of overlooking several unresolved problems still present in current clinical practice. This letter is a call for action on crucial open issues that remain nowadays an unmet need in the management of CNS patients.
Keywords:
crigler najjar;
najjar syndrome;
future make;
looking future;
make forget;
syndrome looking
Journal Title: Orphanet Journal of Rare Diseases
Year Published: 2024
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Journal Title: Orphanet Journal of Rare Diseases
Year Published: 2024
Link to full text (if available)
Share on Social Media: Sign Up to like & get
recommendations!