Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Archives

PTC Therapeutics Provides Update on EMA Regulatory Review Timeline For Its AADC Deficiency Gene Therapy Program

Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency, Clinical Trials, Committee for Medicinal Products for Human Use (CHMP), European Union, Gene Therapy, R&D

PTC Therapeutics Inc. announced that the company completed the Scientific Advisory Group and Oral Explanation meetings for its gene therapy treatment for AADC deficiency (PTC-AADC) with the Committee for Advanced Therapies (CAT).

Ohio State Researchers Use Gene Therapy to Treat Children with Rare AADC Disease

Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency, Children, Clinical Studies, Gene Therapy, Genetic Diseases, Nature Communications, R&D, Rare Diseases, Scientists, The Ohio State University Wexner Medical Center

A study found potential in a novel gene therapy method to help children born with a rare genetic and neurodegenerative disorder, AADC deficiency.

PTC Therapeutics to Acquire Agilis Biotherapeutics

Acquisitions, Angelman Syndrome, Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency, Biologics License Application (BLA), Biotech, Business, CNS, Gene Therapy, Monogenic Diseases, Neurodegenerative Diseases, Neurological Disorders, Product Pipelines

PTC Therapeutics Inc. announced an agreement to acquire Agilis Biotherapeutics Inc., a biotechnology company advancing an innovative gene therapy platform for rare monogenic diseases that affect the central nervous system.