First Scientific Advice obtained from EMA
MYOCURE partners Marinee Chuah (VUB), as coordinator of the MYOCURE project, Kirsty Harper (Envigo), as toxicology expert and Marta Rayo Lunar and Joel Montane Mogas (ASP), as regulatory affairs experts, participated in the first Scientific Advice meeting with the European Medicines Agency (EMA). The meeting was arranged in order to ask for guidance regarding the further design and elaboration of two prototype adeno-associated vectors (AAV) for the treatment of two severe orphan diseases that affect children, the glycogen storage disease II (GSDII, or Pompe disease) and the myotubular myopathy (MTM). Ultimately, the MYOCURE goal is to obtain the orphan status for both products and diseases.
The meeting had a friendly atmosphere and the result has been very positive providing a good foundation for future work in MYOCURE.