Choroid plexus

Human medicines European public assessment report (EPAR): Refixia, nonacog beta pegol, Date of authorisation: 02/06/2017, Revision: 5, Status: Authorised

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Martedì, Gennaio 2, 2024
Chills, Patient, Medical Subject Headings, Marketing, Lethargy, European Commission, Agency, INN, Allergy, Stinger, ATC, Antibody, Restless, Blood, Bleeding, International, PEG, European, Headache, Haemophilia B, Choroid plexus, Ageing, Nausea, Brain, Language, Profile, Swelling, Haemophilia, Flushing, Combustion, Select, Safety, Anatomy, Medicine, Vomiting, IIA, CHMP, Medical device

Human medicines European public assessment report (EPAR): Refixia, nonacog beta pegol, Date of authorisation: 02/06/2017, Revision: 5, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Refixia, nonacog beta pegol, Date of authorisation: 02/06/2017, Revision: 5, Status: Authorised

Lysogene Reports Additional Positive Biomarker Data With LYS-SAF302 and Favorable Safety Data With LYS-GM101 Presented at the WORLDSymposium™ 2022

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Venerdì, Febbraio 11, 2022
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, CNS, Patient, GLB1, LYS, Lists of diseases, Biomarker, Nerve, Financial Services and Markets Authority (Belgium), Company, GM3, Neurodegeneration, Autism, Spinal cord, HIV disease progression rates, GM2, Brain, Forward-looking statement, Review, MPS, Serum, Data monitoring committee, Clinical trial, Safety, HS, Infant, Adaptive clinical trial, GM1, A-DNA, Glycogen storage disease, Fragile X syndrome, Central nervous system, Disease, Blood, CSF, Ganglioside, Choroid plexus, Pharmaceutical industry, Medical imaging, Lysogenic cycle

Data were presented in oral presentations at the WORLDSymposium 2022, on February 10th, 2022.

Key Points: 
  • Data were presented in oral presentations at the WORLDSymposium 2022, on February 10th, 2022.
  • Following treatment with LYS-SAF302 in AAVance, mean serum NF-L concentrations increased, reaching about 2-fold above baseline at 6 months.
  • We look forward to confirming these results in additional patients and timepoints, said Dr. Ralph Laufer, Chief Scientific Officer of Lysogene.
  • Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS).