Advocacy intelligence hub — real-time data for patient organizations
Kebilidi: FDA approved
treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency
Shanghai Vitalgen BioPharma Co., Ltd. — PHASE1
Shanghai Jiao Tong University School of Medicine — EARLY_PHASE1
PTC Therapeutics — PHASE2
Krzysztof Bankiewicz — PHASE1
Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
FINANCIAL LANDSCAPE SUMMARY
Total programs
Open now
Patient Assistance Programs1
Kebilidi
PTC Therapeutics, Inc.
Kebilidi
(eladocagene exuparvovec-tneq)Orphan drugPTC Therapeutics, Inc.
12.1 Mechanism of Action KEBILIDI is a recombinant adeno-associated virus serotype 2 (rAAV2) based gene therapy designed to deliver a copy of the DDC ...
Browse all Aromatic L-amino acid decarboxylase deficiency news →
Yin-Hsiu Chien, MD.,PhD
National Taiwan University Hospital
Krystof Bankiewicz, MD, PhD
OSU Professor of Neurological Surgery
Wuh-Liang Hwu, M.D., Ph.D.
National Taiwan University Hospital
Helio van der Linden
Centro de Reabilitação Dr Henrique Santillo
Katharina Buesch
Global Health Economics and Outcomes Research
Shin-Ichi Muramatsu
Jichi Medical University
View all Aromatic L-amino acid decarboxylase deficiency specialists →