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FDA

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Today we approved an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. Kebilidi is the first FDA-approved gene therapy for treatment of AADC deficiency. https://lnkd.in/er5tacke

  • Graphic indicating FDA approval of the first gene therapy for aromatic L-amino acid decarboxylase deficiency. Features abstract molecular designs and the FDA logo on a blue background.
Kaye Spratt

1h

This is great

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PRATHAMESH VAIDYA

Registered Pharmacist GPAT 2024- 186. NIPER JEE 2024- AIR 336, (AIR-74 in Gen-EWS). BVCET 2024 AIR 56. Bachelor's of Pharmacy- 8.10 CGPA 2024. Diploma in Pharmacy- 88% 2021.

20h

This is great 💐💐

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Rosie Rice

Currently seeking Public-private partnerships and other innovative mechanisms for research are concentrating on neglected diseases in order to stimulate the development of vaccines, drugs and diagnostic

20h

Excellent work

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Jack Zhang

Biologist/Program manager

1d

congratulation

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Santhosh Kumar

Non-clinical, regulatory strategy and risk assessment

46m

Excellent work!

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Thomas Echaniz

Chief Financial Officer

13h

This is great

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Nilesh Kharche, Ph.D

Site Quality Assurance Head

1d

Exciting

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Jacqueline Keller

1d

👏🙏❤️

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1 Reaction
Marie Brace, MBA

Rare Disease Patient Advocate

1d

Congratulations PTC! It’s been a long time coming!

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Pawan Bhatt

PD Downstream Manager GenscriptProBio plasmid purification

19h

Great news! Hoping that growth phase should begin at PTC.

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