Aprobación acelerada de la terapia génica Genglycos (pariglasgene brecaparvovec-opnr) para tratar la enfermedad de almacenamiento de glucógeno tipo Ia (GSDIa) por parte de la FDA.

FDA Approves First Therapy for Patients aged 8 years and older with Glycogen Storage Disease Type Ia Innovative gene therapy offers new option for children and adults with rare disease https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-patients-aged-8-years-and-older-glycogen-storage-disease-type-ia?utm_medium=email&_hsenc=p2ANqtz--x5NKVOSlokwBqiMV1j3riaiZ5IVQrI8ToeRsm19Jd5oUe5fgr8sz4ndgz6C9YOB41ZCesHZ7L06xoqdYQnzXivUS2EQ&_hsmi=144267033&utm_content=144270294&utm_source=hs_email