"Eladocagene exuparvovec"

Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency Highly specialised technologies guidance Published: 19 April 2023 www.nice.org.uk/guidance/hst26 © NICE 2023. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of-rights).Your discrimination, to advance equality of opportunity and to reduce health inequalities. Commissioners and providers have a responsibility to promote an environmentally sustainable health and care system and should assess and reduce the environmental impact of implementing NICE recommendations wherever possible. Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency(HST26)© NICE 2023

Eladocagene exuparvovec (Upstaza) - aromatic L-amino acid decarboxylase (AADC) deficiency 1 Published 11 September 2023 1 SMC2586 eladocagene exuparvovec solution for infusion (Upstaza®) PTC Therapeutics Ltd 04 August 2023 The Scottish Medicines Consortium (SMC) has completed its initial disorder associated with debilitating symptoms, significant impairment in normal motor development milestones, and a high risk of death in young children. Eladocagene exuparvovec is the first medicine licensed for severe AADC deficiency.  In three open-label, single-arm studies in children with severe AADC deficiency, eladocagene exuparvovec treatment resulted

Eladocagene Exuparvovec for Aromatic L-Amino Acid Decarboxylase Deficiency. This JAMA Insights discusses the US Food and Drug Administration approval of Kebilidi, eladocagene exuparvovec, for the treatment of aromatic L-amino acid decarboxylase deficiency in adult and pediatric patients.

Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency. Aromatic L-amino acid decarboxylase deficiency results in decreased neurotransmitter levels and severe motor dysfunction. Twenty-six patients without head control received bilateral intraputaminal infusions of a recombinant adeno-associated virus type 2 vector containing the human aromatic L-amino acid decarboxylase gene (eladocagene exuparvovec) and have completed 1-year evaluations. Rapid improvements in motor and cognitive function occurred within 12 months after gene therapy and were sustained during follow-up for >5 years. An increase in dopamine production was demonstrated by positron emission tomography and neurotransmitter analysis. Patient symptoms (mood, sweating, temperature, and oculogyric

A Study of SmartFlow® Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants This study will have a trial phase, extension phase, and optional long-term extension phase. The primary objective of the trial phase is to assess the safety of the SmartFlow® MR Compatible Ventricular Cannula for administering eladocagene exuparvovec to pediatric participants with aromatic L-amino acid decarboxylase (AADC) deficiency. The extension phase is designed to capture additional clinical information for eladocagene exuparvovec, including changes in motor development, AADC-specific symptoms, and other pharmacodynamic (PD) measures. The optional long-term extension phase is designed to capture long-term safety and efficacy data from participants

• Abemaciclib and other CDK 4/6 inhibitors: an add-on itherapy n certain breast cancers • Eladocagene exuparvovec in AADC deficiency • Prescribing cascades: recognise them and take corrective action • When to take a safety signal into account: Prescrire's experience  > Subscribe now1 December 2023¯¯¯¯¯¯¯¯¯¯¯¯¯¯¯¯¯¯¯¯¯¯¯¯¯Covid-19: Follow Prescrire's independent, evidence-based analysis of the pandemic COVID

of Upstaza (eladocagene exuparvovec) signifies an important landmark, numerous challenges remain. Future research will need to focus on defining the optimal therapeutic window for clinical intervention, better understanding of the duration of therapeutic efficacy, and improved brain targeting. © 2023 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson

Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number): Tai CH, Lee NC, Chien YH, Byrne BJ, Muramatsu SI, Tseng SH, Hwu WL. Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency. Mol Ther. 2022 Feb 2;30(2):509-518. doi

, Chien YH, Byrne BJ, Muramatsu SI, Tseng SH, Hwu WL. Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency. Mol Ther. 2022 Feb 2;30(2):509-518. doi: 10.1016/j.ymthe.2021.11.005. Epub 2021 Nov 8. Responsible Party: National Taiwan University Hospital ClinicalTrials.gov Identifier: NCT02926066