Novel Drug & Emergent Therapeutics | Pharma | Rare Diseases | Inborn Errors of Metabolism
FDA approves brain injection for L-amino acid decarboxylase (AADC) deficiency
Published on MedED: 18 November 2024
Originally published: 24 September 2024
Source: FDA Press Release
Type of article: Novel Drug & Emergent Therapy News
MedED Catalogue Reference: MNDR003
Category: Rare Diseases
CrossreferenceInborn Errors of Mebolism
Category tags: RD, AADC, Injectables, gene therapy
| Product Category | Product Name | Company | Status |
|---|---|---|---|
| FDA Approved |
14 November 2024, FDA Press Release
Treated patients were compared to historical data from untreated individuals. By week 48 post-treatment, motor milestone assessments were available for 12 of the 13 participants. Improvements in gross motor function were observed in 8 of these 12 treated patients, a milestone not seen in untreated patients with severe AADC deficiency.
Access Original Press Release
14 November 2024 | FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency
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