A position statement on the post gene-therapy rehabilitation of aromatic I-amino acid decarboxylase deficiency patients.

Journal Information

Full Title: Orphanet J Rare Dis

Abbreviation: Orphanet J Rare Dis

Country: Unknown

Publisher: Unknown

Language: N/A

Publication Details

Subject Category: Medicine

Available in Europe PMC: Yes

Available in PMC: Yes

PDF Available: No

Transparency Score
3/6
50.0% Transparent
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Evidence found in paper:

"Declarations Ethics approval and consent to participateNot applicable. Consent for publicationNot applicable. Competing interestsREP and JW declare that they have no competing interests. AK and JT are employees of PTC Therapeutics, Inc. H-ML, SM-A, GH, and DM have served as advisory board members for PTC Therapeutics, Inc. W-LH has served as an advisory board member, consulted for, and received research grants from PTC Therapeutics, Inc. Competing interests REP and JW declare that they have no competing interests. AK and JT are employees of PTC Therapeutics, Inc. H-ML, SM-A, GH, and DM have served as advisory board members for PTC Therapeutics, Inc. W-LH has served as an advisory board member, consulted for, and received research grants from PTC Therapeutics, Inc."

Evidence found in paper:

"Funding Funding for this article was provided by PTC Therapeutics, Inc."

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Open Access
Paper is freely available to read
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Tool: rtransparent

OST Version: N/A

Last Updated: Aug 05, 2025