Publication: Intraputaminal gene delivery in two patients with aromatic L-amino acid decarboxylase deficiency

This case study published in Movement Disorders describes the outcomes of 2 patients with aromatic L-amino acid decarboxylase (AADC) deficiency treated with intraputaminal gene therapy

Learn more about intraputaminal gene therapy for AADC deficiency, a rare autosomal recessive disease
Review the key efficacy outcomes of eladocagene exuparvovec treatment in 2 patients over 10 years of age with severe forms of AADC deficiency
Discover the safety profile of eladocagene exuparvovec for these cases

François-Heude MC, et al. Mov Disord Clin Pract. 2023. Online ahead of print.

▼ Eladocagene exuparvovec is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC at pharmacovigilance@ptcbio.com

Eladocagene exuparvovec (Upstaza^TM) is a gene therapy product indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of AADC deficiency with a severe phenotype, currently licensed in the European Union, Great Britain, Iceland, Liechtenstein and Norway. Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The European Summary of Product Characteristics for eladocagene exuparvovec is available here. Please refer to your local country guidance for more information.

Once registered, you will be able to view the abstract and be provided with the DOI and PubMed links for this publication.

Register now to unlock the content

Already registered?
If not, register below

Registration

Firstname *

Surname *

Hospital *

Work email *

Specialty *

Country *

This Website is intended only for healthcare professionals (“Website“), is operated by PTC Therapeutics. (together with its affiliates “PTC”). In order to access the restricted content on this Website:

*

I confirm that I am a licensed healthcare professional and would like to access this Website

*

I understand that PTC will process my personal data, including contact details, for the purposes of providing me access to this Website

I want PTC to provide, by email, updates about new content available to view on MEDhub
PTC will delete immediately the information provided if we cannot confirm you are healthcare professional.
Otherwise, in case of successful verification, PTC will retain the information provided for a period of two (2) years two (2) years following your registration (i) only for the purposes of providing you access to this Website; and/or (ii) in case you signed up to receive PTC updates.

Providing your personal data and your consent is voluntary, and you have the right to withdraw your consent at any time, without giving any reasons. Please consult our Privacy Statement for more information on the processing of your personal data.
For any enquiry relating to the processing of your personal data or if you want to exercise your privacy related rights or withdraw your consent, you can contact PTC’s Privacy Office at: Dataprivacy@ptcbio.com.

You can unsubscribe from updates at any time when receiving an email from PTC.

Publication: Intraputaminal gene delivery in two patients with aromatic L-amino acid decarboxylase deficiency

Authenticating...

COMING SOON